National Institute of Neurological Disorders and Stroke (NIH), USA

2015

  • A drosophila model for studying mechanisms of Gaucher’s disease and synucleinopathies
  • A fly model of autosomal-dominant adult-onset neuronal ceroid lipofuscinosis (ANCL)
  • A molecular anatomic imaging analysis of tau in progressive supranuclear palsy
  • A new antibody/RNAI combination therapy strategy for ALS
  • A novel stat3-selective inhibitor for medulloblastoma therapy
  • A synthetic vesicle to carry modifier compounds to axonal blocks induced by pathogenic POLYQ
  • ALS clinical trials guidelines
  • Amino acid transport and the biology of human gliomas
  • An agonist antibody to musk as a theraputic for musk myasthenia gravis
  • Antigens for molecularly targeted vaccines for progressive glioma
  • Antisense-oligonucleotide-directed inhibition of nonsense-mediated MRNA decay
  • Astrocytes contribution to tuberous sclerosis pathology
  • Autophagy regulation of glioblastoma tumorigenesis and responses to therapy
  • Axonal transport HTS platform for neurodegeneration drug discovery
  • Biomarker discovery for juvenile neuronal ceroid lipofuscinosis
  • Biomarkers of therapeutic response in myotonic dystrophy
  • Blood-based assays for the detection of glioblastoma RNA biomarkers
  • Brain dysfunction in neurofibromatosis
  • Canavan disease: suppressing the phenotype by inhibiting naa synthesis
  • Cellular and molecular studies of SBMA neuromuscular disease
  • Cerebellar function in dystonia
  • Cerebral malaria: mechanisms of disease and neurological salvage
  • Characterization of agrin/lrp4 antibody-positive myasthenia gravis
  • Characterization of an endogenous gaba-ergic mechanism underlying hypersomnia
  • Characterization of class ii pi3-kinase as a homeostatic synaptic plasticity gene
  • Chimeric anti-fibulin-3 reagents for targeted therapy of glioblastoma
  • Commercialization of a diagnostic test for amyotrophic lateral sclerosis (ALS)
  • Comprehensive synaptome proteomics targeting protein expression and PTMS in HD
  • Conditional deletion of MAPT in the adult mouse brain
  • Connexins and glioblastoma progression
  • Coordinate actions between methyl-CPG binding proteins in neuronal development
  • Cortical circuit dysfunction in fragile x syndrome
  • Cortical organization of action knowledge before and after brain surgery
  • Cortical tuber and epilepsy
  • Defining motor neuron-specific MIRNAS in ALS
  • Defining the mechanisms of dipeptide repeat protein toxicity in c9orf72 ALS/FTD
  • Defining the role of FUS phosphorylation in neurodegeneration
  • Determination of the epigenetic regulation of gene transcription by mecp2 in neurons
  • Determining the cellular and molecular basis of mitochondrial encephalomyopathy seizures
  • Determining the role of FOXO in tdp-43 toxicity
  • Development of a new ALS mouse model that targets profilin1 and actin filaments
  • Development of intrasegmental vagus motor neuron subtype identity in Digeorge syndrome
  • Development of molecular therapy for carbamoyl phosphate synthetase deficiency
  • Direct regulation of extracellular proteostasis by the unfolded protein response
  • Disease-modifying genes in Huntington’s disease
  • Does tdp-43 dysfunction in astrocytes trigger motor neuron degeneration?
  • Dopamine neurotransmission in a model of dopa-responsive dystonia
  • Drug discovery for x-linked adrenoleukodystrophy
  • Effect of abcd1 upon brain endothelium in x-linked adrenoleukodystrophy
  • Endothelial plasticity in glioma vascularization and therapy resistance
  • Engineered microenvironments as biomimetic culture platforms for studying the role of brain extracellular matrix in acquisition of resistance to EGFR inhibition across multiple biological scales
  • EOM satellite cells for DMD therapy
  • Epigenetic pathology and therapy in Huntington’s disease
  • Epigenetics in neurodegenerative disease: targeting histone modifications in ALS
  • Etiological linkage of DNA damage/repair deficiency in neurodegenerative diseases
  • Evaluating AAV-mediated gene replacement for spinal muscular atrophy with respiratory distress 1
  • Evolution of pathologic RNA-protein aggregates in motor neuron disease
  • Exploring the contribution of astrocytes to Huntington disease
  • Extrinsic mechanisms governing injury-induced axon degeneration
  • Fabry disease in the GLA knockout rat: development of novel protein therapeutics
  • FMRP and pumilio co-regulate synaptogenesis by controlling neuroglian expression
  • Focus on dicer inhibition as a novel treatment for brain tumors
  • Function & regulation of hippo pathway effectors yap/taz during brain development
  • Functional analysis of ankle2 in microcephaly using a genetic model system
  • Functional characterization of a causative gene for intellectual disability
  • Functional characterization of the GNAQ somatic mutation causing Sturge Weber syndrome
  • Gene delivery for fukutin-related protein deficiencies.
  • Genetic evaluation of the p53 cell death pathway in spinal muscular atrophy (SMA)
  • Genome editing in hd ips cells to reduce mutant and total Huntington expression
  • Glial huntingtin and neurodegeneration
  • Glioblastoma phenotype modulation through MIRNA control of WNT signaling
  • Glioma-astrocyte vascular interactions
  • Glycolytic regulation of cerebellar development and medulloblastoma tumorigenesis
  • G-quadruplex structures as targets and tools in ALS
  • Hdl-loaded nanoparticles for glioma therapeutics
  • Health endpoints and longitudinal progression in congenital myotonic dystrophy (help-CDM)
  • High-bandwidth wireless interfaces for continuous human intracortical recording
  • Host factors and bacterial meningitis
  • How is wallerian degeneration accomplished in the absence of ccr2+ infiltrating macrophages?
  • HPA-axis dysfunction in Huntington’s disease
  • HTS to identify small molecules to disrupt abnormal huntingtin interactions in HD
  • Human EGFRVIII-specific bite for the treatment of glioblastoma
  • Human IPSC and glial chimeric modeling of Pelizaeus-Merzbacher disease
  • Hypoxia regulates notch turnover in glioma stem cells through vasorin
  • Identification of pathogenic mechanisms important in multiple system atrophy
  • Identification of therapeutic compounds for Charcot-Marie-Tooth disease type 1e/2
  • Identifying deficits in brainstem respiratory circuits during viral encephalitis
  • Identifying susceptibility factors for prion diseases
  • Identifying the mechanisms of neuronal fate commitment during direct conversion
  • Image-guided stem cell therapy of perinatal asphyxia in a large animal model
  • Imaging regional gene expression variability in brain tumor associated seizures
  • Immune-suppressive myeloid cells in the glioma microenvironment: signaling mechanisms and novel therapeutic strategies
  • Immunobiology of malignant glioma
  • In vivo analysis of TRKB signaling during sympathetic nervous system development and neuroblastoma pathogenesis
  • Intra-arterial gene therapy for LINCL
  • Intranasal sulfamidase delivery to the brain
  • Investigating cd47-sirpa as novel protective signals during CNS synaptic pruning
  • Investigating disease mechanisms in c9orf72-linked ALS/FTD
  • Investigating glial interactions across the motor exit point during development
  • Investigating mechanisms of ubqln2-mediated neurodegenerative disease
  • Investigating the contributions of astrocyte gap junctions to ALS disease progression
  • Investigating the mechanism and effect of disease-associated increases in the huntingtin long 3’utr isoform
  • Investigating the response of FUS/TLS to excitotoxic insult
  • Investigating v1 interneuron subtype diversity using embryonic stem cells
  • Linking defects in cortical network activity with altered sensory perception in fragile x mice
  • Longitudinal assessment of brain structure and function in myotonic dystrophy
  • Magnetic resonance imaging and spectroscopy biomarkers for Facioscapulohumeral muscular dystrophy
  • Mechanisms mediating NMJ dennervation
  • Mechanisms of c9orf72 hexanucleotide repeat expansion caused neurodegeneration in ALS and FTD
  • Mechanisms of c9orf72-associated dipeptide toxicity
  • Mechanisms of frontotemporal dementia like behavior in progranulin deficient mice
  • Mechanisms of mitochondrial damage in ataxia-telangiectasia
  • Mechanisms of RRAS regulation of huntingtin and turnover
  • Mechanisms to suppress polyglutamine-dependent toxicity in Machado-joseph disease
  • Mechanisms underlying excitability regulation of motoneuron types in ALS
  • Metabolic aberrations in glioma initiation
  • Metabolic control of immune suppression in gliomas
  • Metabolic profiling of sporadic ALS patients: from fibroblasts to neurons and back
  • Methyl-cpg-dependent transcription factor function in human glioma
  • MIRNAS at the c. Elegans neuromuscular junction: potential SMA modifiers
  • Modeling synaptic vulnerability of a brainstem sensorimotor circuit in a mouse model for amyotrophic lateral sclerosis
  • Modeling the effects of reducing huntingtin and HDH alternative splicing in mice
  • Modeling the neurovascular unit using huntington’s disease IPS cells
  • Modeling the progression of sod1-linked motor neuron disease
  • Molecular analysis of torsina function and dysfunction
  • Molecular and cellular mechanisms of Miller-Dieker syndrome
  • Molecular control of brain size
  • Molecular control of myelinating glial cell development by fbxw7
  • Molecular genetics of SCA1
  • Molecular mechanism of pid1, a novel tumor inhibitor, in glioblastomas
  • Molecular mechanisms of huntingtin misfolding
  • Mouse model of progressive multifocal leukoencephalopathy
  • Mouse pneumotropic virus infection: a model for JC virus latency and reactivation
  • Mtor substrate phosphorylation: a new bioassay for therapeutics
  • Multiple system atrophy – novel targets in early diagnosis, pathophysiology, and therapeutic approach
  • Muscle stem cells: new ALS growth factor therapy and disease model
  • Myoferlin in muscle membrane fusion and repair
  • Myotubularin pi 3-phosphatases as regulators of peripheral nerve myelination
  • Nad metabolsim and mitochondrial dysfunction in ALS models
  • Nad, pgc-1alpha and sirt3 as therapeutics targets for Huntington’s disease
  • Nanocarriers designed to deliver nucleic acids to brain
  • Neurodevelopmental outcomes in children with severe malaria
  • Neuroinflammatory effects on striatal ca2+-permeable ampa and kainate receptors
  • New tools for computing macro and microscopic channel properties from structure
  • Next generation electrical impedance myography: the virtual muscle biopsy
  • Nonmuscle myosin IIA and the glioma phenotype
  • Novel mechanisms of actin dynamics underlying cell motility, axon growth, and ALS
  • Nuclear mechanisms of polyglutamine toxicity in SBMA
  • Nucleocentric molecular characterization of c9orf72 NRE-linked neurodegeneration
  • Nurture: research training and mentoring program for career development of faculty at makerere university college of health sciences
  • Optimization of a novel series of thiazolopyridines for the treatment of SMA
  • Optimizing correction of the Huntington’s disease mutation by trans-splicing
  • Pathogenic mechanisms of c9orf72 GGGGCC repeat expansions in amyotrophic lateral sclerosis and frontotemporal dementia and development of therapeutic strategies
  • Patholog-omics – essential tremor in the broader context of neurodegeneration
  • Pathophysiology of upper and lower limb motor control in spinocerebellar ataxia
  • Pharmacological chaperone therapy for the gm2 gangliosidoses
  • Phenotypic effects of brain-directed enzyme therapy for Sanfilippo B syndrome
  • PPST-transcriptional regulation of gene expression in neuromuscular disease
  • Precision medicine for pediatric low-grade gliomas
  • Preclinical evaluation of a novel combinatory epigenetics based therapy for glioblastoma
  • Protective role of neuregulin-1 against cerebral malaria pathogenesis and mortality
  • Protein succination as a mediator of neuropathology in mitochondrial disease
  • Rare disease research training program
  • Re-establishing vascular integrity in ALS via endothelial cell transplant
  • Regulation of glutamate transport in astrocyte subtypes and in ALS
  • Regulation of mitochondrial function and motor neuron degeneration in SMA
  • Reprogramming the human glioma genome
  • Resources for studying neural circuit structure and function with g-deleted rabies viruses
  • RNA binding proteins in complex neurological disease
  • RNA dysregulation in neurodegeneration
  • RNA processing defects in SMA and their contribution to the disease phenotype
  • Role of a novel troy-EGFR complex in gliobastoma invasion and resistance
  • Role of NNOS cortical neurons in slow wave activity production and cognition
  • Safety and efficacy of systemic gene therapy in informative models for DMD
  • Seeded transmission of SOD1 misfolding
  • Signaling pathways regulating oligodendrocyte development and function
  • Simple-regulated trafficking and peripheral neuropathy
  • Spinal muscular atrophy: inducing SMN expression: administrative supplement
  • Stat3 and astrogliosis in Alexander disease
  • Statistical methods for causal inference in observational studies
  • Stem cell-based therapy for Canavan disease
  • Studies of NMJ structure and function in mutant FUS-ALS and beyond
  • Synaptic calcium dysregulation in Huntington’s disease
  • Targeted delivery of a sonic hedgehog inhibitor for the study of medulloblastoma therapeutics
  • Targeted pharmacological chaperones for neurological diseases
  • Targeting bet-bromodomains in neuroblastoma
  • Targeting histone demethylase activity for the treatment of pediatric brainstem glioma
  • Targeting olig2 co-regulators for malignant glioma therapy
  • Targeting prmt5 in cancer initiating cells as a new therapy for glioblastoma
  • Targeting the chaperone pathway for myelin repair in hereditary neuropathies
  • Targeting tumors with NF1 loss
  • Targeting wee1 in MYC driven medulloblasoma
  • Tau conformation in tauopathies and neuronal function
  • Tdp-43 aggregation inhibitors for the treatment of ALS
  • Thalamocortical circuit defects in developmental brain disorders
  • The contribution of cytoskeleton disruptions in ALS disease pathology
  • The development of small molecule inhibitors for Gaucher disease type 3
  • The nature of astrocyte heterogeneity in RTT
  • The role of glycyl-trna synthetase in axonal health and disease
  • The role of h3k27m-induced aberrant prc2 activity in brainstem gliomagenesis
  • The role of kinesin motors in actively delivering cargos into dendritic spines
  • The role of nde1 in the pathogenesis of microcephaly and schizophrenia
  • Therapeutic modulation of myotonic muscular dystrophy
  • Therapeutically relevant targets of twist1 dimers in glioma
  • Transcriptional control of glioma development
  • Translation of AAV p0 ice schwannoma gene therapy to clinical trials
  • Transposable elements in frontotemporal lobar degeneration
  • Treatment of ALS based on transplantation of glial restricted progenitors
  • Underlying molecular mechanisms of gliogenesis and gliomagenesis in the central nervous system
  • Untargeted metabolite profiling of sporadic ALS patient fibroblasts: identifying mechanisms of the disease
  • Upstream open reading frames in neuronal function: a singular and genome-wide approach
  • Using the c. Elegans oocyte to model the cell biology of early onset dystonia
  • Vesicular ca2+ channels in sarcolemma repair and muscular dystrophy
  • Visual circuit regression and its rescue in RTT mouse models
  • White matter vessels in cerebral malaria
  • Whole body single AAV microgene therapy in canine DMD

2014

  • A longitudinal analysis stream for freesurfer
  • A model of TRPV4 channelopathy
  • A mouse model to define immunovirologic determinants of polyomavirus CNS disease
  • A novel role for a bicistronic calcium channel gene in neurodevelopment and neuro
  • A novel xenograft model of FSHD
  • Acetate supplementation as a therapeutic strategy for Canavan disease
  • Alk signaling in CNS development, ischemic brain injury and gliomagenesis
  • Antisecretory factor as a novel therapeutic agent in human glioblastoma
  • Antisense oligonucleotides to treat spinal and bulbar muscular atrophy
  • Assessing interactions between cerebrovascular and tau pathologies
  • Aurora-a is a novel therapeutic target in glioblastoma
  • Autosomal dominant FTLD tauopathy patient-specific stem cell models
  • Biomarkers of brain permeability in human cerebral cavernous malformations
  • Braf mutation in malignant astrocytoma origin, evolution, and response to therapy
  • Brain MRI and histopathology of the epileptic baboon
  • Brain tumor targeting using tumor-specific neuroimmunology
  • Brain-penetrating nanoparticle therapeutics for invasive brain cancer
  • C9orf72 in motor system biology and ALS
  • C9ran proteins as a biomarker for c9orf72-associated ALS and FTD
  • Can klotho alleviate muscle fibrosis in muscular dystrophy?
  • Ccr4 in regulatory t cell trafficking to glioma
  • Cellular and molecular mechanisms of FUS-related amyotrophic lateral sclerosis
  • Cellular and molecular mechanisms regulating glial growth control and survival
  • Cellular mechanisms of fatal respiratory insufficiency in arboviral encephalitis
  • Centrosome regulation and function associated with microcephaly
  • Cerebellar involvement and alcohol effects in a mouse model of myoclonus dystonia
  • CGG repeat associated translation in fragile x-associated tremor/ataxia syndrome-diversity supplement
  • Characterization of white matter in Huntington’s disease using diffusion MRI
  • Citrulline protects against cerebral malaria by reducing metabolic encephalopathy
  • Complement-microglia interaction in synaptic loss and neurodegeneration in HD
  • Contextual glioblastoma screening for efficacious radiation sensitizers
  • Creation and correction of spinal muscular atrophy in the pig
  • CSF1 signaling in gliomagenesis
  • CSF-enhanced-aggregation biomarker for Huntington’s disease
  • Cyclic epha4 peptide antagonists for neuroprotection in ALS
  • Defining an electrophysiological phenotype of ALS patient-derived motor neurons
  • Determining the hierarchy of signaling pathways regulated by the CCM1 protein
  • Developing ASO therapy for repeat expanded c9orf72 ALS-FTD
  • Development and in vitro validation of therapy for Mucopolysaccharidosis III
  • Development of a mouse model of c9orf72 ALS/FTD expressing ran translated peptide
  • Development of a mouse model of progressive multifocal leukoencephalopathy
  • Development of a multi-modal neuroimaging biomarker for amyotrophic lateral sclerosis
  • Development of an antisense oligonucleotide therapy for SOD1 familial ALS
  • Development of an information-rich assay for c9orf72 as a test for ALS and FTD
  • Disease modifying potential of glatiramer acetate in Huntington’s disease
  • Disordered regulation of wnt/beta-catenin signaling in MPNST development and maintenance
  • Dissecting molecular and genetic mechanisms that promote myelination
  • Diversity supplement for MYCN, MTOR and translation control in medulloblastoma
  • Dual-vector mediated mini-dystrophin restoration of function in mdx model of DMD
  • Dynamin-related protein 1, neurodegeneration and Huntington’s disease
  • Elucidating signaling pathways for Gaba-a receptor alpha5 in an aggressive subtype of medulloblastomas
  • Endolysosomal function in neuronal maintenance
  • Enzyme replacement therapy for GM1 gangliosidosis lysosomal rare disease
  • Ephrin ligands as novel targets for an adjunct therapy in cerebral malaria
  • ERAD genes that suppress neurodegeneration
  • Establishment of a human grade IV glioma stem cell line bank
  • Exploring a role for line1 retrotransposons in neurodegenerative disease
  • Factors modulating the interaction of Huntingtin with lipid membranes: implications for Huntington’s disease
  • Functional dissection of the k27m histone mutation in vivo
  • Gene delivery for fukutin-related protein deficiencies
  • Generating IPSC-derived neurons to explore formation & inhibition of human prions
  • Generation of intermediate neural progenitors in drosophila larval brains
  • Genetic analysis of copper toxicity mechanisms in IPSC-derived human neurons
  • Genetic modifiers of Duchenne muscular dystrophy
  • Genetic modifiers of phosphoinositide mediated neurodegeneration
  • Glioma induced alterations in neuronal transcription and translation
  • Gpr56 is a regulator of glial cell development and myelination
  • Hematopoietic stem cell-based therapy for Friedrich ataxia
  • High-throughput screening for Nad+-replenishing neuroprotective compounds
  • Huntingtin proline-rich region modulation of Huntington’s disease pathogenesis
  • Huntington’s disease biomarkers and therapeutics
  • Identification of endophenotypes in the behavioral-variant of Frontotemporal Dementia
  • Identification of therapeutic compounds for Charcot-Marie-Tooth disease type 1E/1
  • Identification of transcription factors that regulate astrocyte differentiation
  • Identification of TSC cellular phenotypes using patient-derived IPSCS
  • Identifying the second hit in MYC-driven medulloblastoma; a role for GFI proteins
  • Il13ra2 targeted t-cell therapy for glioma
  • Imaging of hippocampal activity across sleep/wake and disease states
  • Imaging of protein synthesis and ubiquitination in Fragile X syndrome
  • Immunological biomarker studies in myasthenia gravis
  • Immunotherapy ameliorate neurological deficits in encephalitis
  • Improving MPS I ERT efficacy through lectin-mediated delivery
  • In vivo longitudinal assessment of methylene blue for Huntington’s disease
  • Inflammatory mediators and mechanisms during central nervous system infection
  • Inhibitory microcircuitry coordinates striatal function
  • Interactions between tdp-43 and microrna-92 in drosophila and human neurons
  • Interferon regulation of the blood brain barrier during West Nile encephalitis
  • Interplay of the microbiome and the brain in neurodegenerative disease
  • Intranasal stem-cell based therapy for glioblastoma
  • Investigating the pathogenesis of cerebellar neurodegeneration
  • Investigating the role of mecp2 in activity-dependent gene expression
  • Investigation of apicct1 for Huntington’s disease therapeutics
  • Investigation of solute transport from the cerebrospinal fluid to CNS
  • LNCRNA regulation of the HTT gene via a hominid-specific transposable element
  • Longitudinal evaluation of familial frontotemporal dementia subjects (LEFFTDS)
  • Manganese enhanced MRI: non-invasive measure of pathogenesis of dystrophinopathy
  • Mapping central and peripheral metabolomic markers in Huntington’s disease
  • Mass cytometry analysis of signaling dysfunction in Duchenne muscular dystrophy
  • Mechanisms and effects of GABABR dephosphorylation during ischemic excitotoxicity
  • Mechanisms and rescue of neural circuit dysfunction in mecp2 mutant mice
  • Mechanisms of CGG ran translation in fragile x-associated tremor/ataxia syndrome
  • Mechanisms underlying Joubert syndrome related brain malformations
  • Metabolic approaches to Friedreich ataxia
  • Metabolism informs intertumoral & intratumoral heterogeneity
  • Misfolded ALS-linked profilin-1: a novel therapeutic target
  • Mitochondrial antioxidant neurodegenerative therapeutics: xjb-5-131 derivatives
  • Modeling neural development using human IPSCS from TSC patients
  • Modulation of FCRN: a strategy to prevent autoantibody-mediated nerve injury
  • Molecular basis of hyperhomocysteinemia induced brain injury in ischemic stroke
  • Molecular mechanisms of neuroprotection in polyglutamine-dependent degeneration
  • Molecular mechanisms of peripheral prion pathogenesis
  • Molecular neuroimaging of disease mechanisms in cerebral amyloid angiopathy
  • Molecular pharmacology of the system xc- glutamate/cystine antiporter
  • Movement and sensation in cerebellar ataxia
  • MYCN, MTOR and translation control in medulloblastoma
  • Myotubularin pi 3-phosphatases as regulators of peripheral nerve myelination
  • Neuroregulatory mechanisms of pias1 and implications for Huntington’s disease
  • New nonsense suppression drugs to treat MPS I
  • Novel adoptive transfer therapy for glioma using car-transduced type17 t-cells
  • Novel combinatorial therapies for malignant peripheral nerve sheath tumors
  • Novel CSF diagnostics and genotype markers of tuberculosis meningitis in zambia
  • Novel epigenetic targets for silencing the Huntington’s disease mutation
  • Novel mechanisms for distal transport in developing and mature neurons
  • Optogenetic dissection of striatal circuits in a mouse model of human dystonia
  • Pathobiology of neurodegeneration in c9orf72 repeat expansion
  • Pathway-specific nf-kappab regulatory networks in glioma
  • Pediatric low-grade gliomas: biology and molecular targeting.
  • Pet imaging of hyperphosphorylated tau differentiates PSP and CBD from PD
  • Polo-like kinase 1 as a therapeutic target in medulloblastoma
  • Preclinical evaluation of NMDA receptor antagonists for treating Rett syndrome
  • Ras proteins in nerve tumorigensis
  • Redesign rat model for ALS research
  • Regulation of dendritic kv4.2 expression by neuronal microRNAS
  • Regulation of pmp22 expression in peripheral nerve
  • Regulation of protein targeting in axon guidance and neuronal morphogenesis
  • Rescue of gabaergic function in a mouse model of Rett syndrome
  • RNA lariat debranching enzyme as a novel drug target
  • Role and therapeutic value of AHR in inflammatory macrophages during GBM
  • Role of ezh2 in medulloblastoma tumorigenesis
  • Role of lap positive immune cells in glioblastoma pathogenesis
  • Role of p62 in amyotrophic lateral sclerosis
  • Role of the blood brain barrier in neurologic sequelae
  • Silencing c9or72 with raav mediated RNAI
  • Small chemical modulators of SMN biology as candidate therapeutics for SMA
  • Spinal muscular atrophy therapy using recombinant SMN proteins
  • Structural correlates of prion strain, phenotype and infectivity in human prion d
  • Sulfamidase brain drug delivery in Sanfilippo syndrome
  • Synaptic origins of cerebellar disease
  • Synthetic biomarkers of RNA modulation therapies
  • Targeting the novel pi5p4k pathway to induce glioblastoma senescence
  • Tau conformation in tauopathies and neuronal function
  • Tdp-43 acetylation as a pathogenic modification in ALS & related proteinopathies
  • Tdp-43 and mitochondrial dysfunction in ALS
  • Testing the role of small RNAS in FMR1 promoter silencing in Fragile x syndrome
  • The circuit basis for rapid disinhibition during whisker map plasticity in rodent somatosensory cortex
  • The frontotemporal lobar degeneration clinical research consortium
  • The perkplexity of the integrated stress response in motor neurons and myelinatin
  • The prion protein as a probe for identifying neurotoxic protein oligomers
  • The relationship between tau pathology and sleep in tauopathies
  • The role of bk channels in neuropathology of fragile x syndrome
  • The role of chemokine signaling in sickle cell pain
  • The role of kif9 in glioma invasion
  • The role of microRNAS in epilepsy of tuberous sclerosis complex
  • The role of phosphor-rps6 and translational regulation in glioma
  • The role of sirtuin 1 deacetylase in amyotrophic lateral sclerosis
  • The role of tau in the neuropathology of cerebral malaria
  • Therapeutic targeting of aberrant glial function during juvenile batten disease
  • Therapeutics of MTOR signaling in succinic semialdehyde dehydrogenase deficiency
  • Therapy for leptomeningeal medulloblastoma by a novel implantable pump
  • Toward drug treatment of spinal muscular atrophy: mechanism of action
  • Transcription therapeutics for myotonic dystrophy type 1
  • Truncated gli1 in glioblastoma
  • Using cell-penetrant peptides to target atf5 in mouse glioma models
  • Using patient-derived neurons for epilepsy drug discovery
  • Using rna signatures for therapy development in neurodegeneration due to c9orf72 expansions
  • Using tsunami in a sensitized mouse to generate an intermediate SMA model
  • Validating a novel target for correction of pathophysiology in Fragile x and TSC
  • Validation of novel pathogenic HTT post-translational modifications (PTMS)

 

2013

  • A gene and progenitor cell therapy in Huntington disease mice (website)
  • A Knockin Mouse Model for Oculopharyngeal Muscular Dystrophy (website)
  • A Multi-Modal MRI/PET Study of Parkinsonism: Diagnosis and Disease Progression (website)
  • A Next Generation of Biomarkers for Incipient Huntington Disease (website)
  • A Phase II Trial of Rituximab In Myasthenia Gravis (website)
  • A system for in vivo structure-function analysis of kinesin in neurodegeneration
  • A Visualization Tool for Computer-Aided Diagnosis of Focal Cortical Dysplasia (website)
  • Adaptive EdU Therapy for Brain Tumors (website)
  • Adult Neurological Phenotypes of Fragile X Gray Zone Expansion (website)
  • Alfy-mediated selective macroautophagy and the pathogenesis of Huntington’s Disease (website)
  • All optical control and monitoring of neural activity (website)
  • ALS/FTD mutant C9orf72-induced genetic and nuclear pathology in iPS cell models (website)
  • Alterations in Blood-Brain/Blood-Spinal Cord Barrier Properties in Amyotrophic (website)
  • Analysis of autophagy and mitochondrial homeostasis in a human iPS model of NCL (website)
  • Analysis of the RNA-binding Protein FUS and Its Role in Neurodegenerative Disease (website)
  • Analysis of the tumor suppressor gene Tsc1 in motor neuron patterning (website)
  • Antisense oligonucleotides for the treatment of spinocerebellar ataxia type 2 (website)
  • Astroglial Orexin in Sleep Disorders (website)
  • Autoimmune mechanisms in a novel Aire-deficient model of peripheral neuropathy (website)
  • Blocking TLR-Activation of Regulatory T cells Slows Disease in ALS (website)
  • Brain Network Imaging: A Novel Biomarker for Preclinical Huntington’s Disease (website)
  • Brain tumors with regulatory T-cells treated with EGFRvIII-specific T-cells (website)
  • Cellular and Molecular Mechanisms of FUS-related Amyotrophic Lateral Sclerosis (website)
  • Characterization of a novel translational model for Becker muscular dystrophy (website)
  • Characterizing the role of specific Ras proteins in neurofibroma and MPNST format (website)
  • Cockayne syndrome: role of the innate immune response in neurodegeneration (website)
  • Convergent Synaptic Mechanisms in Neurodevelopmental Disorders (website)
  • Co-regulation and function of Pur-gamma and WRN in the CNS (website)
  • Determining the role of IRE1alpha in amyotrophic lateral sclerosis (website)
  • Development of a HTS Assay for a Neurological Lysosomal Disease (website)
  • Development of a Porcine Model of Juvenile Neuronal Ceroid Lipofuscinosis (website)
  • Development of SLIC, a methodology for synthetic lethal screening in the CNS (website)
  • Development of Zebrafish Taupoathy Models for Translational Research (website)
  • Dissecting the genetic underpinnings of essential tremor (website)
  • Dissecting the in vivo role of Huntingtin in Rab vesicle movement on microtubules (website)
  • Dissecting the role of cellular transport in neurodegeneration using A.nidulans (website)
  • Distal effects of botulinum neurotoxins (website)
  • Dopamine D1 Receptor in mouse models of primary dystonia (website)
  • Dynamic Magnetic Targeting of Activated Brain Macrophages for Glioma Therapy (website)
  • Effect of ALS-causing Mutations on Binding of TDP43 and FUS to RNA in iMNs (website)
  • EGFRvIII-targeted Bispecific T cell Engagers for brain tumors (website)
  • Elucidating and understanding the genetic basis of movement disorders (website)
  • Elucidating pathological mechanism of DI-CMTC (website)
  • Endoplasmic reticulum quality control of mutant HexA enzyme in Tay-Sachs disease (website)
  • Enzyme Replacement Therapy for Sanfilippo A Lysosomal Rare Disease (website)
  • Epigenetic mechanisms of TDP43-mediated neurodegeneration (website)
  • Epigenetic Regulation of Neuronal Cell Death (website)
  • Epitope-Specific Targeting of Tau Aggregates (website)
  • Evaluating HMG-CoA reductase as a therapeutic target in ALS (website)
  • Evaluating the efficiency and specificity of stop codon suppression therapy (website)
  • Evaluating the role of NRG-1 receptors in MPNST tumorigenesis (website)
  • Evaluation of the in vivo effects of genes regulating Ataxin1 levels in SCA1 mice (website)
  • FMRP-regulated association of specific miRNAs with RISC (website)
  • Frontotemporal Dementia Induced Pluripotent Stem Cell Consortium (website)
  • FSHD iPS Cells: Genetic Correction and Myogenesis (website)
  • FTD risk factor TMEM106B alters endolysosomal function and progranulin pathways (website)
  • Functional analysis of the huntingtin N17 domain knock-in mouse models (website)
  • Galectin modulation of glutamate receptors and neuronal function
  • Gaucher disease:Treatment of neurodegenerative disease (website)
  • Gene delivery for fukutin-related protein deficiencies (website)
  • Gene delivery to muscle and nerve for laminin-alpha2-deficient MD (MDC1A) (website)
  • Gene Discovery in Muscular Dystrophy (website)
  • Gene discovery in PSP by transcriptome, neuropathology and sequence analysis (website)
  • Gene regulatory mechanisms that assign and maintain motor neuron terminal differe (website)
  • Generation of Tau Prion Strains in Dividing Mammalian Cells (website)
  • Generation of TWIST1 reporters through characterization of TWIST1 dependent netwo (website)
  • Genetic Control of Neural Stem Cell Homeostasis (website)
  • Genetic Correction of a Novel « Knock-in » Mouse Model for Farber Disease (website)
  • Genetic Determinants of Tauopathies (website)
  • Genetic labeling and visualization of CSMN in models of motor neuron disorders (website)
  • Genetic modifiers of Predict-HD phenotypes (website)
  • Genetics and biology of a viable mutant mouse with dystonic movements (website)
  • Genetics and Biology of CIZ1 in Cervical Dystonia (website)
  • Genomic and functional analysis of transcriptome changes in Huntington’s Diseasea (website)
  • Genomic Disorders in Neurodevelopmental Disease (website)
  • Glioma-Astrocyte Vascular Interactions (website)
  • Granulin specific monoclonal antibodies to investigate their expression and role (website)
  • GSK3 regulation of suprachiasmatic neuronal excitability and light entrainment (website)
  • High Content Sceening for Hereditary Stroke Syndrome (website)
  • High throughput screen for selective inhibitors of cyclophilin B (website)
  • Highly specific, temporally controllable mouse genetic tools for investigating in (website)
  • Huntington’s disease biomarkers and therapeutics (website)
  • Identification of Amyotrophic Lateral Sclerosis Clinical Predictors (website)
  • Identification of Immune modulators associated with JC virus replication (website)
  • Identification of mutation causing Purkinje cell degeneration in the shaker rat (website)
  • Identifying symptomatic and neuroprotective strategies for cerebellar ataxia (website)
  • Identifying the Role of PrPSc Distribution & Other Proteins in Prion Pathogenesis (website)
  • Imaging and Genetics in Huntington’s Disease (website)
  • Imaging biomarkers of the FTD-ALS spectrum (website)
  • Imaging Circuit Change in the Motor Cortex of Mouse Model of ALS (website)
  • Immortalized Human Strital Precursors as a Cell Model of HD (website)
  • Imprinting defects leading to Angelman and Prader Willi syndromes (website)
  • Improving Diagnostics and Neurocognitive Outcomes in HIV/AIDS-related Meningitis (website)
  • In vivo magnetic resonance-based analysis of inherited neurologic disease after g (website)
  • Inhibiting glioma invasion using targeted nanoparticles (website)
  • Integrated approach to protein biomarker identification in Huntington Disease (website)
  • Intrathecal Gene Therapy Expressing IGF-1 for Amyotrophic Lateral Sclerosis (website)
  • Investigating cell-type-specific contribution to JNCL (website)
  • Investigating the molecular basis of Canavan disease (website)
  • iPS Cells for Investigation of HDL2 and HD Pathogenesis (website)
  • Katanin p80: A Role for Regulation of Microtubule Severing in Cortical Developmen (website)
  • Knock-in Mice with Spinocerebellar Ataxia Type 13 Mutations in Kcnc3 (Kv3.3) Gene (website)
  • Local Proliferation of Glia and their Interaction with Blood Vessels (website)
  • LRP4 signaling in neuromuscular junction formation (website)
  • Matrix Metalloproteinases: Therapeutic Targets For Huntington’s Disease (website)
  • Mechanism of Small Molecule Mediated Rescue of Mytonic Dystrophy (website)
  • Mechanism of SPARC peptide FSEC inhibition of angiogenesis in neuroblastoma (website)
  • Mechanisms and synergies of therapeutic drug treatments in muscular dystrophy (website)
  • Mechanisms of cell contact inhibition and their dysregulation in cancer
  • Mechanisms of glioma growth and invasion novel therapeutic strategies (website)
  • Mechanisms of Neurodegeneration in ALS (website)
  • Mechanisms Underlying the Cerebellar Contribution to Autism in Mouse Models of Tu (website)
  • Mechanistic studies of transthyretin misfolding and amyloid formation through a c (website)
  • Metabolite Profiling of a Novel Mitochondrial Kinase in Neuromuscular Disease (website)
  • Modeling Mutant Profilin 1 Toxicity and ALS in vivo (website)
  • Modeling spinal cord axis patterning with human pluripotent stem cells
  • Molecular assembly and regulation of the cerebral cavernous malformation complex (website)
  • Molecular genetic studies of progranulin regulators in FTLD and ALS (website)
  • Molecular Mechanisms Underlying Glioma Invasion of the Human Subventricular Zone (website)
  • Molecular Signatures of Amyotrophic Lateral Sclerosis in Skeletal Muscle (website)
  • Motor neuron based reporter screening assay for spinal muscular atrophy (website)
  • Motor neuron diversity: markers, regulatory mechanisms, and functional relevance
  • Mouse Models to Define Critical Periods and Molecular Targets in FXTAS (website)
  • MRI Biomarkers of Patients with Tuberous Sclerosis Complex and Autism (website)
  • MRI-based mapping of regional genomic diversity in Glioblastoma (website)
  • mtDNA heteroplasmy in development and differentiation: an in-vitro approach (website)
  • mTOR modulation of myelination (website)
  • NanoGenomics to study molecular mechanisms of neurodegeneration in human cells (website)
  • Neural Stem Cell Mediated CE-CPT11 Therapy for Neuroblastoma (website)
  • Neuroactive Steroid GABAA Receptor Positive Modulators for Fragile X Syndrome (website)
  • Neuroimaging and Neuropathology of Mucopolysaccharidosis I (website)
  • Neuronal autophagy inducers as an approach to treat diseases of the central nervo (website)
  • Neuronal specific Autophagy Signaling (website)
  • Neuroprotective drugs for A-T (website)
  • Next generation gene silencing strategies for Huntington’s disease (website)
  • Novel adhesion mechanisms in glioblastoma stem cells (website)
  • Novel high-throughput screening for modifiers of TorsinA pathology (website)
  • Novel mechanistic study of CMT2D neuropathy (website)
  • Novel monocyte effector function in CLL immune therapy (website)
  • Novel small-molecule inhibitors of Wee1 kinase for medulloblastoma treatment (website)
  • Nuclear export of RNAs by nuclear envelope budding (website)
  • Optimization of HDAC6 Inhibitors in the Treatment of CMT (website)
  • Optimizing ring screening strategy for control of taenia solium infection
  • Origin of Cortical Hyperexcitation in MeCP2-deficient Brain (website)
  • Overcoming barriers in the study of in vivo spinal cord function (website)
  • Oxysterol Biomarkers For Niemann-Pick C Disease (website)
  • Patterning dendritic branches with environmental and neuronal surface molecules (website)
  • Phagocytosis is misregulated in a Drosophila model of Fragile X syndrome (website)
  • Phase II Trial of SGS-742 in Succinic Semialdehyde Dehydrogenase Deficiency (website)
  • Physiological significance of persistent inward currents in motor neurons (website)
  • Pilot study of preclinical and prodromal frontotemporal degeneration (website)
  • Placode lineage contribution to Hirschsprung’s disease (website)
  • Polarized transport in nervous system development and disease in zebrafish
  • Polycomb and Cellular Hierarchy in the Brain (website)
  • Post-transcriptional regulation of gene expression in neuromuscular disease
  • Posttranslational regulation of augurin, a new secretory tumor suppressor
  • Preclinical Treatment for NF2-Associated Tumors (website)
  • Rare and Novel Genetic Variation in the Pathogenesis of Cerebellar Ataxia (website)
  • RBC-derived microparticles function in cerebral malaria (website)
  • Reductive Stress in Complex I Deficiency (website)
  • Respiratory motor neuron protection following cervical spinal cord injury
  • Reversal of CMV-specific Immune Deficits in Patients with Glioblastoma (website)
  • RNA Dysfunction in Selectively Vulnerable Populations in SCA7 Mice (website)
  • RNA foci in C9FTD/ALS (website)
  • RNA-Gain-of-Function Pathogenesis in SCA10 (website)
  • Role of IRF4-NOTCH2 Interplay in B-Cell Development and Malignancy (website)
  • Role of MMP-9 in selective motor neuron degeneration in ALS (website)
  • Role of Neural Progenitor Cells in the Development of Neonatal Hydrocephalus (website)
  • Role of Parkinson’s disease gene PARK2 in GBM oncogenesis and genomic instability (website)
  • Role of Target-derived FGFs in Maintaining and Repairing Synapses (website)
  • Role of the Huntington’s Disease Protein in Post-Transcriptional Gene Silencing (website)
  • Selectively inhibited transcription of mutant genes in neurodegenerative disease (website)
  • Sexually dimorphic miR-497 regulates alpha-synuclein and alpha-synucleinopathy (website)
  • Site-directed RNA editing: a new method to correct disease causing mutations
  • Small Molecule p75 Neurotrophin Receptor Ligand to Treat Huntington’s Disease (website)
  • Small molecule screen to suppress expression of mutant Huntington
  • STAT3 Regulation of Glioblastoma Pathogenesis (website)
  • Studying a novel regulator of GBM tumorigeneis: the role of CK2alpha in maintaini (website)
  • Synaptic Contribution to Disease in Mouse Models of Charcot-Marie-Tooth (website)
  • Synaptic regulation of mTOR-dependent protein synthesis at synapses (website)
  • TAAR1 Agonists as Narcolepsy Therapeutics (website)
  • Targeting MELK-mediated EZH2 signaling in glioma stem cells (website)
  • Targeting TAK1 in Neuroblastoma (website)
  • Targeting the cholesterol metabolism to treat glioblastoma (website)
  • The amelioration of peroxisomal disorders due to defects in Pex10 (website)
  • The COPA vesicle protein and pathogenesis of spinal muscular atrophy (website)
  • The Gateway Hypothesis: A new framework for unraveling diverse leukodystrophies (website)
  • The Genetic Regulation and Disease Function of the Frontotemporal Dementia Protei (website)
  • The interaction of IDO and Tregs Leads to Immunosuppression in Glioma (website)
  • The Role of PGC-1aplha in the Pathogenesis of Myotonic Dystrophy Type 1 (website)
  • The Role of TDP-43 Phosphorylation in Protein Function and Neurodegeneration (website)
  • The Role of the Terminal Complement Pathway in ALS (website)
  • The survival of motor neuron protein in axonal mRNA localization (website)
  • TorsinA function and dystonia-related dysfunction in developing and mature CNS (website)
  • Toward understanding disease pathogenesis in the polyglutamine disorder Spinocere (website)
  • Tracking the in vivo proliferative history of human glioma-derived stem cells (website)
  • Transcranial Stimulation in Spino-Cerebellar Ataxia (website)
  • Translating a CSF delivered AAV9-SMN for treatment of Spinal Muscular Atrophy (website)
  • TSC-mTOR on neuron development (website)
  • Tumor cell and microenvironment changes causing antiangiogenic therapy resistance (website)
  • Understanding the cellular basis of Movement Disorders (website)
  • Understanding the Mechanisms of TDP-43 Function (website)
  • Understanding the Pathogenic Mechanisms of Rett Syndrome (website)
  • Using High Field MRI to Evaluate Metal Dyshomeostasis in Huntington’s Disease (website)
  • Validation of ubiquilin for Huntingtons disease (website)
  • Virus nanoparticles as autophagy activators (website)
  • Wild-type nerve grafting promotes reinnervation of SOD1 muscle (website)

 

2012

  • 4D Shape Analysis for Modeling Spatiotemporal Change Trajectories in Huntington’s (website)
  • 5-Hydroxymethylcytosine in Neurodevelopment (website)
  • A Dose-Escalation, Safety and Feasibility Study Of Enteral Levetiracetam For Seiz (website)
  • A hybrid microvesicle/virus vector for targeted gene transfer to the brain (website)
  • A Large Animal Model of SMA (website)
  • A Model System to Study Human Neuromuscular Disease (website)
  • A novel bioengineered technique to rapidly and permanently repair cut PNS nerves (website)
  • A Novel Model of Medulloblastoma to Define Cancer Pathways and Molecular Targets (website)
  • A Novel Xenograft Mouse Model of Facioscapulohumeral Muscular Dystrophy (website)
  • Absorption, Metabolism and Biodistribution of Prions after Oral Ingestion (website)
  • Active zone loss cause dying back neuropathy in amyotrophic lateral sclerosis (website)
  • Advancing Gene Therapy for Late Infantile Neuronal Ceroid Lipofuscinosis (website)
  • Aerobic glycolysis regulates apoptosis in neurogenesis and medulloblastoma (website)
  • Allele-Specific RNAi to treat Genetic Prion Disease – Resubmission 01 (website)
  • Alpha M Beta 2 integrin blockade for acute inflammatory neuropathies (website)
  • Alterations In Adult Neurogenesis During West Nile Virus Encephalitis (website)
  • An Animal Model of LRSAM1 Peripheral Neuropathy (website)
  • Analysis of a novel duplication locus causing human cerebellar malformation (website)
  • Antisense oligonucleotide knock-down of ataxin-7 in a SCA7 mouse model (website)
  • Autoimmune biomarker profiling in tauopathy (website)
  • Automating MRI Delta T1 Methods for the Routine Assessment of Brain Tumor Burden (website)
  • B7-H1 Expressing Macrophages Mediate Immunosupression in Glioma (website)
  • Basal Ganglia Shape Analysis and Circuitry in Huntington’s Disease (website)
  • Blockade of p53 and Aurora A in Therapy Resistant Neuroblastoma (website)
  • BRAF Mutation in Malignant Astrocytoma Origin, Evolution, and Response to Therapy (website)
  • Brain Cancer Stem Cell Reprogramming by c-Met (website)
  • Brain endothelial cell function under adenosine receptor signaling directive (website)
  • Brain tumor restricts developmental potential in intermediate progenitor cells (website)
  • Calcium channels as novel therapeutic targets for Huntington’s Disease (website)
  • Cavities in Choline Acetyltransferase and Neuromuscular Disorders (website)
  • Cell Type Specific Tracing of Neocortical Circuits Using Viral Vectors (website)
  • Cellular and Molecular Consequences of Respiratory Chain Defects in Neurons (website)
  • Cerebellar Function in Health and Disease (website)
  • Cerebellar stimulation for the treatment of dystonia: preclinical studies (website)
  • Cerebello-Basal Ganglia Interactions (website)
  • Chemical Probes Targeting Gliomas with IDH Mutation (website)
  • CNS mechanisms of developmental synapse elimination (website)
  • CNS Tuberculosis (website)
  • Congenital brain malformations caused by aberrant head mesenchymal signaling (website)
  • Cortical organization of object knowledge before and after brain surgery (website)
  • CRCNS: Propagation of beta oscillations in cortico-basal ganglia-thalamic loop (website)
  • Deciphering mechanisms of perivascular tumor cell invasion in glioblastoma (website)
  • Deciphering RNA based mechanisms of neurodegeneration (website)
  • Defining a clinically relevant time point for astrocyte targeted therapy in ALS (website)
  • Defining the messenger RNP code in the brain (website)
  • Defining the role of brain iron dysregulation in Huntington’s disease (website)
  • Determinants of Neurodegenerative Decline in Primary Progressive Aphasia (website)
  • Developing a microRNA-Targeted Therapy for ALS (website)
  • Developing resources to alleviate muscle atrophy in FSHD by genome engineering (website)
  • Development of a Biochemical Diagnosis for Creutzfeldt-Jakob disease (website)
  • Development of a Novel Biomarker for Mucopolysaccharidosis I, II, and VI (website)
  • Development of a Read-Through Drug for Duchenne Muscular Dystrophy (website)
  • Development of mouse models of optineurin-linked ALS (website)
  • Differential Approach to the Postural Tachycardia Syndrome (website)
  • Disease Mechanisms in Human Ubiquilinopathy (website)
  • Dissecting the preB?tzinger complex neural network using holographic photolysis (website)
  • Dopamine Neurotransmission in Tourette Syndrome (website)
  • DUX4 inhibition as a therapeutic strategy for FSHD (website)
  • Early Biomarkers of Autism Spectrum Disorders in infants with Tuberous Sclerosis (website)
  • Effect of early psychosine accumulation in Krabbe Disease on CNS progenitor cells (website)
  • Effect of NF1 mutation on choroid plexus function (website)
  • Effects of PolyQ Expansion on Full-length Huntingtin Protein in HD (website)
  • Elucidating the cellular mechanisms of prion propagation and clearance for devisi (website)
  • Energy Balance and Neurodegenerative Disease (website)
  • Examination of the earliest symptoms and biomarkers of FTLD MAPT carriers (website)
  • Exploring Disease-Toxicant Interactions in a Human Huntington’s Disease Model (website)
  • Function of the Sigma-1 Receptor in Motoneurons (website)
  • Functional characterization of astrocytes in a mouse model of Rett syndrome (website)
  • Functional Connectivity in Premanifest Huntington’s Disease (website)
  • Functional Role of Micro RNAs in Huntington’s Disease Pathogenesis (website)
  • GAA Repeats Induced Epigenetic Silencing in Friedreich’s Ataxia (website)
  • Gene discovery in primary dystonia using whole exome sequencing (website)
  • Generation and Characterization of Amyotrophic Lateral Sclerosis (website)
  • Generation and Characterization of Novel Drosophila Models of TDP-43 Toxicity (website)
  • Generation of DYT1 dystonia-specific iPS cells with isogenic controls (website)
  • Genetic and Functional Profiles of Serotonergic Neuron Subtypes (website)
  • Genetic Modifiers of Immune Evasion by Cytomegalovirus in Glioblastoma (website)
  • Genome analysis to elucidate molecular mechanisms for peripheral neuropathies (website)
  • Genome-wide screening for effectors of toxic prion protein-induced neuronal death (website)
  • Genomic Studies in Charcot-Marie-Tooth Disease (website)
  • Genotypic Interactions in Brain Cancer Heterogeneity (website)
  • GGGGCC hexanucleotide repeat expansions in neurodegenerative disease (website)
  • Glycosylation-independent enzyme therapy of the brain in Sanfilippo B syndrome (website)
  • Guanabenz in the treatment of mutant SOD1 ALS mice (website)
  • Heparan sulfate proteoglycans as critical regulators of brain cancer malignancy (website)
  • High Throughput Screening for Compounds to Mitigate Toxicity of FUS/TLS &SOD1 (website)
  • High-affinity RNA targets of Survival Motor Neuron Protein (website)
  • Huntington’s Disease: Analysis of Proteolysis (website)
  • Identification and regulation of quiescent stemcells in the adult brain (website)
  • Identification of genes responsible for sarcolemmal integrity in C. elegans (website)
  • Identification of novel system xc- inhibitors (website)
  • Implications of a novel glioblastoma classification defined by miR-21-Sox2 status (website)
  • In vivo and crude extract analysis of polyQ aggregation intermediates (website)
  • In vivo proof of efficacy studies for a novel glucosylceramide synthase inhibitor (website)
  • Influence of Anti-Angiogenic Therapy on Drug Delivery to Brain Tumors (website)
  • Innate immunity in Rett pathology and repair (website)
  • Investigating a Toxic Gain-of-Interaction Between FUS/TLS & Stress Granules (website)
  • In-vitro brain organotypic model of Progressive Multifocal Leukoencephalopathy (website)
  • Is xenon neuroprotective in a mouse model of ALS (website)
  • Isoform-specific effects of MeCP2 isoforms on neuronal viability (website)
  • Large scale language networks Topography and selective degeneration (website)
  • Liposome-siRNA-Peptide Complexes as Therapy to Cure Prion Diseases in Mouse Model (website)
  • Live-Cell Assays for Lysosomal Enzyme Activity (website)
  • LRRK2 modulates the progression of neuropathology in a mouse model of tauopathy (website)
  • Magnetic-vortex microdiscs for glioma therapy (website)
  • Mechanism Underlying Neurodegeneration Caused by Dysfunction of Pre-mRNA Splicing (website)
  • Mechanisms of Caspase-1 Mediated Schwannoma Regression (website)
  • Mechanisms of Central Synaptic Dysfunction in SMA (website)
  • Mechanisms of degeneration in the spinal cord and musculoskeletal system in SMA (website)
  • Mechanisms of prion spread (website)
  • MeCP2 regulation on LINE-1 retrotransposition in neurons (website)
  • Merlin regulation of spinal cord NSC function (website)
  • Microparticles, a new player in cerebral malaria pathogenesis (website)
  • Mitochondrial Porin in Bioenergetic Defects in Huntington’s Disease (website)
  • Mitochondrial RNA transport as a novel therapy (website)
  • Modulating aggrephagy to modify Huntington’s disease (website)
  • Modulation Of Cellular Clearance To Treat Human Disease (website)
  • Modulation of Lipid Metabolism to Rescue Aberrant Synaptic Transmission in HD (website)
  • Modulation of lysosomal function for the treatment of neuronal ceroid lipofuscino (website)
  • Modulation of the PPAR-delta Signaling Pathway in Huntington’s Disease (website)
  • Molecular and cellular characterization of SMN-mediated U7 snRNP assembly (website)
  • Molecular Basis of Iron Imbalance in sCJD Brain and CSF (website)
  • Molecular basis of Scapuloperoneal SMA and Charcot-Marie-Tooth disease type 2C (website)
  • Molecular Mechanisms of CTIP2 Function in Corticospinal Motor Neuron Development (website)
  • Molecular Mechanisms of IDH1-mediated Gliomagenesis (website)
  • Molecular Motors and Glioma Dispersion (website)
  • Multiple Roles Of FMRP In Synaptic Function And Plasticity (website)
  • MyoSense: Automated Muscle Hypertonicity Classification System (website)
  • Nemo-Like Kinase and the Pathogenesis of Spinal and Bulbar Muscular Atrophy (website)
  • Neurobiological Predictors of Huntington’s Disease (website)
  • Neuronal regulation of myelination (website)
  • Non-invasive Markers of Neurodegeneration in Movement Disorders (website)
  • Novel approaches to study microglia physiology and pathology in the intact brain (website)
  • Novel gene therapy strategies for Canavan disease (website)
  • Novel therapy for monoamine neurotransmitter deficiency in PKU (website)
  • Optimization of compounds to improve mRNA splicing in familial dysautonomia (website)
  • Optogenetic control of striatal dopamine in Huntington’s disease (website)
  • Optogenetic dissection of striatal circuits in a mouse model of human dystonia (website)
  • Optogenetic modulation of episodic dystonia in the tottering mouse (website)
  • Orexin Receptor Agonists for the Treatment of Excessive Daytime Sleepiness and Ca (website)
  • Osteopontin-mediated invasion and dissemination in central nervous system lymphom (website)
  • Overcoming therapeutic resistance of gliomas (website)
  • Pathogenesis of a JC Virus Variant in Pyramidal Neurons (website)
  • Pathogenic impact of Huntington phosphorylation in Huntington’s Disease (website)
  • Phase 1b Trial of stem cells in ALS (website)
  • Physiological mechanisms responsible for cognitive impairments in Dravet Syndrome (website)
  • Preferential single-strand break repair in the active genes of mammalian cells (website)
  • Presurgical brain mapping with functional connectivity MRI (website)
  • Primary Prevention of Strokes in Nigerian Children with Sickle Cell Disease (website)
  • Prostaglandin E2 Signaling and Protection from Endoplasmic Reticulum Stress-Induc (website)
  • Protein Aggregation and Inclusion Body Formation (website)
  • Regenbase: A Searchable Database to Organize Regeneration Knowledge via Ontologie (website)
  • Regulation of mTORC1 signaling by mROS and ATM (website)
  • Regulation of neural development by TGF beta family signaling (website)
  • Regulation of Neurogenesis in TSC by mTORC1 and mTORC2 (website)
  • Revealing new regulators of EGFR-P13K driven glioma proliferation and migration (website)
  • Rock Inhibition as Therapy for Cerebral Cavernous Malformation (website)
  • Role of C-type Lectin Receptors in Myeloid Plasticity in Neurocysticercosis (website)
  • Role of ERK1/2 in Neuromuscular Synapses and Myofiber Development in vivo (website)
  • Role of Stasimon Dysfunction in Spinal Muscular Atrophy (website)
  • Selective Activation of Neurons to Control Narcolepsy (website)
  • Self-complementary rAAV9 Systemic Gene Delivery Treatment for MPS Type IIIA (website)
  • SMA Biomarkers in the Immediate Post-natal Period of Development (website)
  • SMA mouse models: splicing manipulation and therapeutic SMN replacement (website)
  • Spinal and brainstem respiratory neurons in Pompe disease (website)
  • Study of a Novel Deubiquitylase in Medulloblastoma (website)
  • Studying the glial contribution to RTT pathogenesis using patient-specific iPSCs (website)
  • Studying The Role Of Inflammatory Mediator MIR-155 In A Mouse Model Of Als (website)
  • SVIP and CMT1A (website)
  • Synaptic signaling in a human stem cell model of Angelman syndrome (website)
  • Targeted Inhibition of NMD to Enhance the Efficacy of Readthrough Drugs (website)
  • Targeting proteostasis to treat Niemann-Pick C disease (website)
  • Targeting Tau Splicing for Dementia (website)
  • TDP-43 acetylation as a pathogenic modification in ALS & related proteinopathies (website)
  • Testing of Innate Immunity Stimulation via TLR9 on CAA using Non-human Primates (website)
  • Testing Therapies in a New Mild SMA Mouse: A Model for Type II/III Patients (website)
  • The effects of the unfolded protein response on medulloblastoma (website)
  • The Functional Role of RBM45 in Gene Expression and Neurodegeneration (website)
  • The HD iPSC Consortium: Repeat Length Dependent Phenotypes for Assay Development (website)
  • The mechanism linking traumatic brain injury to amyotrophic lateral sclerosis (website)
  • The NKCC1 inhibitor bumetanide as a novel therapy in TSC (website)
  • The PDGF alpha neural progenitor cells of the subventricular zone contributes to (website)
  • The role of microglial-and neuron-specific MyD88 signaling in tauopathies (website)
  • The role of mir-17~92 cluster in motor neuron degeneration (website)
  • The role of mTOR signaling in oligodendrocyte differentiation and CNS myelination (website)
  • The role of Sirt1 in Huntington’s Disease (website)
  • The role of spartin in autophagy of lipid droplets: Implications for SPG20 disease (website)
  • The role of the terminal complement pathway in experimental cerebral malaria (website)
  • The toxicity of the RNA CGG repeats in FXTAS (website)
  • Therapeutic Development for Amyotrophic Lateral Sclerosis in a Canine Model (website)
  • Tooth pulp as a source for neuronal precursor cells to study neurogenetic disorde (website)
  • Transcriptome in Huntington’s disease and Huntington’s disease-like 2 (website)
  • Treatment for Dysmyelination in PMD and SPG2 (website)
  • Triplex-mediated DNA repair as a novel therapy for Hurler Syndrome patients (website)
  • Truncated NK1R in GBM: Pharmacology and Relationship with Patient Survival (website)
  • Understanding the role of MAPT in Parkinsonian disorders (website)
  • Understanding the Role of Mitochondrial Import In Amyotrophic Lateral Sclerosis (website)
  • Validation of the HD-HRQOL (Huntington disease quality of life measure) (website)
  • Zebrafish models of muscular dystrophies (website)
  • Zinc-Finger Nuclease- Mediated Repair of Disease-Causing Triplet CAG Repeats (website)

 

2011

  • A device for rapid, painless, bedside muscle evaluation of children (website)
  • A Follow-up of Children Enrolled in the Management of Myelomeningocele Study (website)
  • A New Approach to ALS (website)
  • A Novel Small Molecule Therapy for Tay-Sachs and Sandhoff Diseases (website)
  • Adenosine A2A receptor cross-activation of TrkB in Huntington’s disease (website)
  • Agrin/alpha 3 Na,K-ATPase signaling at the neuromuscular junction (website)
  • Alpha-synuclein downregulation in Spinal Muscular Atrophy (website)
  • Antisense oligonucleotide treatment for myotonic dystrophy (website)
  • Apparatus and Method for Convection Enhanced Therapeutic Delivery (website)
  • Assessment of Neurological Deterioration in Subjects with LINCL (website)
  • Ataxin-2 as a genetic risk factor for ALS: New insights into neurodegeneration (website)
  • Autophagic Clearance of Aberrant Tau: Biochemical and Therapeutic Implications (website)
  • Axonal Transport and RAS Activation in the NF1 mouse model (website)
  • Biophysical and molecular dialogue of glioma cells and the brain microenvironment (website)
  • Brain Delivery of Nanoformulated Superoxide Dismutase (website)
  • Breathing disorders in a mouse model of Rett syndrome (website)
  • Cell Type-Specific Synaptic Defects in Angelman Syndrome Model Mice (website)
  • Cell-autonomous mechanisms of Ube3a function in the neocortex (website)
  • Cellular genes and signaling pathways as therapeutic targets for virus-induced CN (website)
  • Cellular physiology of epilepsy-associated KCNQ2 channels (website)
  • Cerebral Malaria: Mechanisms of disease and neurological salvage (website)
  • CHADSS: Chagas Disease Scan Study (website)
  • Characterization of epsilon-sarcoglycan interacting proteins in mouse brain (website)
  • Characterization of nucleotide-dependent structural rearrangements in atlastin (website)
  • Characterization Of Prion Strains And Infectivity (website)
  • Characterizing the functional stem-like properties of primary and recurrent glioma (website)
  • Cholesterol Homeostasis in Malignant Glioma (website)
  • Clinical Research Sites for the Network of Excellence in Neuroscience Clinical Tr (website)
  • Clustering Postsynaptic Proteins at Neuromuscular Synapses: From Dok-7 to Rapsyn (website)
  • Cognitive Impairment Moderated by Working Memory in Pediatric Partial Epilepsy (website)
  • Complement activation promotes CD133+ glioma cell proliferation (website)
  • Contemporary Therapeutics For Anaplastic Gliomas (website)
  • Conversion of fibroblasts to functional spinal motor neurons using defined factor (website)
  • Crosstalk between glioma cells & immune cells in the tumor microenvironment: Ther (website)
  • Cyclophilin B as a therapeutic target in glioblastoma (website)
  • Cytoprotective mechanism against misfolded SOD1-induced toxicity (website)
  • Defining the role for Gle1 in the fetal lethal motor neuron disease LCCS1 (website)
  • Defining the role of Ataxin-2 in ALS and TDP-43 proteinopathies (website)
  • Defining the role of BRAF in glioma initiation and maintenance (website)
  • Defining the role of mutant IDH in glioma initiation and maintenance (website)
  • Developing A Mouse Retinal Model Of Neurodegenerative Disease (website)
  • Developing UPR Modulators as Novel Therapeutics for Neurodegeneration (website)
  • Development of a Knock-in Mouse Model for Spinocerebellar Ataxia Type 3 (website)
  • Development of a Porcine Model of Ataxia-Telangiectasia (website)
  • Development of an Oral Therapeutic Drug for Spinal and Bulbar Muscular Atrophy (website)
  • Development of anti-DUX4 therapeutics for FSHD (website)
  • Development of Novel Mouse Models to Study REST Function in Medulloblastoma (website)
  • Development of therapeutic fusion inhibitor peptides for Measles encephalitis (website)
  • DP ARF Ultrasound for Monitoring Muscle Degeneration in Duchenne Muscular Dystrop (website)
  • Drug Screening Assays for Charcot-Marie-Tooth Disease (website)
  • DUSP26: A Novel Therapeutic Target in Neuroblastoma (website)
  • Effects of P-188 on Respiratory Function and Diaphragm Degeneration in the mdx mo (website)
  • Effects of Testosterone and Genetic Factors on Psychological and Motor Function I (website)
  • Electrical and mechanical properties of motor units in a mouse model of ALS (website)
  • Electrophysiological phenotypes in a Drosophila model of SMA (website)
  • Elucidating molecular structure of mammalian prions (website)
  • Elucidating the Mechanisms Underlying Mutant TDP43-induced Neurodegeneration (website)
  • Endocytosis in JNCL (website)
  • Enhancement of Proteasome Activity for the Treatment of Neurological Disorders (website)
  • Enzyme replacement therapy of the brain in Mucopolysaccharidosis Type II (website)
  • Epigenetic Markers in Huntington’s disease Brain (website)
  • Epigenetic Regulation of Heterochromatin Condensation in Huntington’s Disease (website)
  • Epigenome Interactions in Complex Neurogenetic Disorders (website)
  • Exploring Therapeutic Misconception and Increasing Wellbeing Related to Clinical (website)
  • Expression reporter mice for non-invasive imaging: PGC-1a and Huntington’s Disease (website)
  • Functional Brain Imaging with Oscillating Gradient DW-MRI (website)
  • Functional Imaging of Tremor Circuits and Mechanisms of Treatment Response (website)
  • FUS/TLS gain and loss of function in ALS: animal and cellular models of disease (website)
  • GABA chaperoning of epilepsy-associated GABAA receptor mutants (website)
  • Gene Expression in Motor Neurons with Differential Susceptibility to ALS (website)
  • Generation of a mouse model for Progressive Supranuclear Palsy (website)
  • Genetic Analysis of EGFRvIII-independent Glioblastoma Maintenance (website)
  • Genetic studies to gain insight into the function of the MeCP2 domains in vivo (website)
  • Genome Studies in Hereditary Spastic Paraplegia (website)
  • Harnessing novel cell-penetrating antibodies for neuronal correction (website)
  • Human Neural Stem Cells for HD: Technical and Empirical Advances (website)
  • Human-specific evolution of GADD45g forebrain expression (website)
  • Identification of compounds that inhibit aggregation and toxicity of TDP-43 (website)
  • Identification of genes causative for familial ALS gene using exome sequencing (website)
  • Identification of Novel X-linked Intellectual Disability Genes (website)
  • Identification of Therapeutic Small Molecules for Myotonic Dystrophy Type 1 (website)
  • Identifying excitotoxic mechanisms in a C. elegans model of axon destabilization (website)
  • Immunotherapy against tauopathy in a transgenic mouse model (website)
  • In vivo characterization of the basal ganglia indirect pathway (website)
  • Increase Rab11 Activity as HD Therapy (website)
  • Inflammasome Activation in Complex Regional Pain Syndrome (website)
  • Interaction of ASIC and BK channels and its role in glial proliferation (website)
  • Interplay between Intrinsic and extrinsic force and glioma pathogenesis (website)
  • Intrathecal cyclodextrin therapy of feline Niemann-Pick type C disease (website)
  • Investigating mechanisms in torsionA proteasomal degradation (website)
  • Investigating the homeostatic role of MeCP2 in mature brain (website)
  • Investigation of Gemin5 in Coordinated mRNA Regulation (website)
  • Investigation of mechanism of action of drug induced agranulocytosis (website)
  • Investigation of the role of the N17 headpiece in huntingtin aggregation (website)
  • KRIT1 in Vascular Development and Dysfunction (website)
  • Linking TAU Filament Structure To Phenotypic Diversity In Human Tauopathies (website)
  • Macrophage, blood-brain barrier, and modulation of neurodegeneration (website)
  • Mechanisms of Chemotherapy Induced Cognitive Defects (website)
  • Mechanisms of Krabbe Disease Pathobiology and Therapy (website)
  • Mechanisms of prion aggregation and species barriers (website)
  • Mechanistic basis for endosomal dysfunction in frontotemporal dementia linked to (website)
  • Membrane properties of neurons controlling jaw function in a mouse model of ALS (website)
  • Misregulation of the TDP-43 RNA target, Sortilin, in neurodegeneration (website)
  • Modeling Apoptotic Suppression in a Mouse Model of Brain Tumors (website)
  • Modeling Gene Regulation Essential for Long-Term Plasticity (website)
  • Modeling Pathogenesis of Huntington’s disease using patient-derived induced pluri (website)
  • Modulating the BBB to Improve Drug Delivery to the Brain (website)
  • Modulation of the blood-tumor barrier through targeted suppression of claudin 5 (website)
  • Molecular Mechanism of Prion Strain (website)
  • Molecular mechanisms linking early life seizures, autism and intellectual disabil (website)
  • Motoneuron-selective Rescue of SMA Model Mice (website)
  • mRNA Targets for TDP-43 and FUS/TLS: Identifying Key RNA-Processing Errors in ALS (website)
  • Na-K-Cl cotransporter in Glioblastoma Multiforme (website)
  • National Brain and Tissue Resource for Parkinson’s Disease and Related Disorders (website)
  • Network of Excellence in Neuroscience Clinical Trial (NEXT) (website)
  • Neural correlates of impaired consciousness in childhood absence epilepsy (website)
  • Neuroaxonal Dystrophy in Purkinje Cell Death in NPC Disease (website)
  • Neurodegeneration and Proteotoxicity Dissected in C. elegans and Mammals (website)
  • Neuronal nicotinic receptor modulation and cerebellar ataxias (website)
  • New Insights into Motor Neuron Disease (website)
  • New Models to Assay Gene Silencing Therapies (website)
  • Next-Generation Sequencing Approaches For Novel Gene Discovery In ALS (website)
  • Nicotinic acetylcholine receptors and neuroblastoma (website)
  • NINDS Network for Excellence in Neuroscience: Clinical Research Site at UC Davis (website)
  • NINDS NEXT: Children’s Hospital Boston Clinical Research Site (website)
  • Noncoding RNAs at the heart of the Prader-Willi locus (website)
  • Novel CXCR4 Therapeutics to Block Bevacizumab-Induced Glioma Dissemination (website)
  • Novel Genetic Mouse Model to Study the Consequences of TorsinA Dysfunction (website)
  • Novel RNAi therapy for ALS and other Neurodegenerative Disorders (website)
  • Novel Therapeutic Agents for Gliomas with IDH Mutations (website)
  • Oligonucleotides that Modulate AMPA Receptor Alternative Splicing as Drug Candida (website)
  • Pathologically modified TDP-43 in neurodegenerative diseases (website)
  • Peptide aptamers against prion proteins as therapeutic and analytic tools (website)
  • Perivascular astrocyte morphology during motor neuron degeneration (website)
  • Phenotyping pain in a mouse model of pancreatic cancer (website)
  • Polarity Signals and MyosinII Coordinate Neuron Migration and Germinal Zone Exit (website)
  • Pooling of Prospective Studies of Diet, Lifestyle and ALS (website)
  • Preclinical model of intra-arterial gene therapy for late infantile neuronal cero (website)
  • Preclinical neuroprotective trials of a PPAR-gamma agonist in HD mouse models (website)
  • Pre-clinical testing of necrostatin as a potential small molecule for the treatme (website)
  • Presenilin dysfunction in the brain (website)
  • Progranulin interaction with Sortilin in FrontoTemporal Dementia (website)
  • Rapid-onset Dystonia Parkinsonism (DYT12 Dystonia): Pathophysiology & Atp1a3 Mice (website)
  • Regulation and function of the vascular niche in glioma recurrance (website)
  • Regulation of ClC-3 in Human Malignant Glioma (website)
  • Regulation of the Vasculature by Invading Glioma Cells (website)
  • Replacement of Motor Neurons and Astrocytes in ALS Using Human Embryonic Stem Cel (website)
  • Replication mechanism of human prions (website)
  • Reprogramming of disease motor neurons (website)
  • Rescuing Niemann-Pick C Disease: Pathways of Liver and Brain Degeneration (website)
  • Rescuing radiation-induced cognitive deficits through cranial transplantation of (website)
  • Revealing protein synthesis defects in Fragile X Syndrome with new chemical tools (website)
  • Reversible conditional models for Huntington’s disease (website)
  • RNAi screen of the glioblastoma stem cell kinome under hypoxia and normoxia (website)
  • RNAi Therapy for Dominant Limb Girdle Muscular Dystrophy Type 1A (website)
  • Role of ABC efflux transporters in ALS (website)
  • Role of Brain Ferroxidases in AD and sCJD Pathogenesis (website)
  • Role of Dopamine Receptors In Primary Focal Dystonias (website)
  • Role of FUS in ALS (website)
  • Role of genome organizer Satb1 in Shh-dependent proliferation and tumorigenesis (website)
  • Role of mTORC2 in GBM – development of a novel therapeutic mTOR kinase inhibitor (website)
  • Roles for TDP-43 and FUS in ALS Using Motor Neurons from Embryonic Stem Cells (website)
  • Signature-based chemical screening for DYT6 dystonia (website)
  • Skeletal Muscle Mitochondrial Dysfunction and Motor Unit Number in Sporadic ALS (website)
  • Small Molecule Probes for TAR DNA Binding Protein 43_TDP-43 (website)
  • Small Oligonucleotides as Therapeutic Agents Of Spinal Muscular Atrophy (website)
  • Splice isoform-specific RNAi as therapy for Spinocerebellar Ataxia type 6 (website)
  • Stem cell mediated targeting of tumor cells and associated vasculature in gliomas (website)
  • Structure-Function of the Nuclear Envelope Bridge and its Role in Laminopathies (website)
  • Structure-Function Relationships in Dystonia: A Network Approach (website)
  • Study on Neurodegeneration Using TDP-43 Transgenic Rats (website)
  • Studying Early-Stage Lesions in Mouse Models of Cerebral Cavernous Malformations (website)
  • Suppression of Glioblastoma Stem Cells by Kruppel-Like Factor 9 (website)
  • Suppression of polyQ proteotoxicity by glutamine rich proteins (website)
  • Targeted Modulation of the Death Receptor as a Therapeutic Strategy for Glioma (website)
  • Targeting Mitochondrial Superoxide in Angelman Syndrome (website)
  • Targeting SREBP-1 regulated fatty acid synthesis pathway to treat glioblastoma (website)
  • The essential role of supporting cells in medulloblastoma tumorigenicity (website)
  • The Histone Demethylase SMCX/JARED1C as a Therapeutic Target for Huntington’s Disease (website)
  • The Identification and Manipulation of RNA Splicing Factor Regulators in Spinal M (website)
  • The impact of post-translational modification on SOD1 aggregation in ALS (website)
  • The in vivo Role of Serine 421 in Huntington’s Disease (website)
  • The mechanism of blood brain barrier impairment in cerebral cavernous malformation (website)
  • The Notch signaling pathway in glioblastoma (website)
  • The role of androgen receptor acetylation in the polyglutamine disease SBMA (website)
  • The Role of Desoxysphingoid Bases in HSAN1 (website)
  • The Role of Huntingtin in Striatal Neurogenesis (website)
  • The role of IDO and Th17 in an experimental mouse model of glioma (website)
  • The Role of Lysosomal Glucocerebrosidase in Synucleinopathies (website)
  • The role of mTORC1 in the development and therapeutic targeting of NF1-associated (website)
  • The Role of the AR N/C Interaction in SMBA (website)
  • The role of the N/C interaction of the androgen receptor in SBMA (website)
  • The vascular niche in glioma propagation and resistance to therapy (website)
  • Toxicity of a SUMOylated Fragment of the Glial Glutamate Transporter EAAT2 in ALS (website)
  • Trafficking and Function of Central Nervous System Resident Regulatory T Cells (website)
  • Transcriptional Control of Adult Neural Stem Cells (website)
  • Treating the CNS and Somatic Diseases of MPS IIIB by Systemic Gene Delivery (website)
  • Tumor-Inhibitory Effects of P1D1 in Gliomas (website)
  • Understanding neuronal and axonal degeneration in a murine model of human MS (website)
  • Unglued Glia, a Seven Transmembrane Protein Essential for Glia Differentiation (website)
  • Using Drosophila as a model to understand TDP-43 function in ALS (website)
  • Validating NT17 Dependent Mechanisms in Huntington’s Disease Suppression (website)
  • Validation of a Disease-Specific Instrument for Pediatric Inherited Neuropathy (website)
  • Validation of IKKbeta as a therapeutic target for HD (website)
  • Validation of structural MRI as a biomarker in Huntington’s disease (website)
  • Vascular Delivery of alpha-Sarcoglycan for LGMD2D (website)

 

2010

  • A Drosophila model to investigate prion-like transmission of Huntington’s disease (website)
  • A Functional Cell-Based Screen for Potential SMA Therapeutic Compounds (website)
  • A Spontaneous Mouse Model of Motor Neuron Disease (website)
  • A zebrafish model for neural regeneration (website)
  • Aberrant activation of mTOR in peripheral neuropathy (website)
  • Aberrant GABAAR Trafficking and Synaptic Inhibition in Huntington’s Disease (website)
  • Acetylcholine Receptor-Mediated Competition for Synaptic Input (website)
  • Advancing a Glioma Immunotherapy (website)
  • ALS-associated TDP-43 Aggregation: A Drosophila Model and A Role for Autophagy (website)
  • Angiogenin in ALS Pathobiology and Therapy (website)
  • ATM (Ataxia Telangiectasia Mutated) signaling through cyclin D1 (website)
  • Axonal Transport Deficits during Hereditary Spastic Paraplegia (website)
  • Biostatistics and Data Management for a Trial of Corticosteroid Regimens in DMD (website)
  • Blood Level of Huntingtin: A Biomarker for Huntington’s Disease (website)
  • CD4+CD25+ Treg cells regulate microglia and provide neuroprotection in ALS (website)
  • Cell-Cell Transfer And Propagation Of Tau Aggregates (website)
  • Cell-specific translational profiling in Huntington’s Disease mouse models (website)
  • Cellular pathways underlying polyglutamine degeneration (website)
  • Cellular regulation by protein ubiquitination/deubiquitination (website)
  • Chaperone-mediated autophagy in spinocerebellar ataxia type 7 (SCA7) (website)
  • Characterization of KIFAP3, a Modifier of Survival in Sporadic ALS (website)
  • Chemical genomic profiling for hereditary dystonia (website)
  • Citrulline adjunctive therapy for cerebral malaria (website)
  • Combining Anti-Invasive and Anti-Angiogenic Therapies for the treatment of GBM (website)
  • Congenital Myasthenic Syndromes: Pathogenic Mechanisms (website)
  • Contribution of AMPK to motor neuron disease (website)
  • Cortical and basal ganglia local field potentials in human movement disorders (website)
  • Cytomegalovirus Gene Expression and Strain Variability in Glioma Pathogenesis (website)
  • Defining the Mechanism Of Tdp-43 Related Neurodegeneration (website)
  • Defining the Role of TDP-43 in Neurodegenerative Disease Pathways (website)
  • Developing in vivo and in vitro Models for ALS Caused by Mutant FUS (website)
  • Developing therapeutic intervention for FXTAS (website)
  • Development and Testing of Hsp90 Inhibitors (website)
  • Development of a novel quinazoline compound for the treatment of spinal muscular (website)
  • Development of Animal Models of Anti-MuSK Myasthenia (website)
  • Development of novel compounds to inhibit PRMT5 enzyme in high grade astrocytomas (website)
  • Discovering More Juvenile Myoclonic Epilepsy Genes by a Consortium (website)
  • Drosophila models of human cognitive disorders: NF1 and Noonan syndrome (website)
  • Early Detection of Cerebral Amyloid Angiopathy (website)
  • EFECAB: Improving pig management to prevent epilepsy in Burkina Faso (website)
  • Emergence of drug resistance in prion populations (website)
  • Exploiting Trans-Suppression to Arrest hSOD Aggregation in ALS (website)
  • FOR-DMD:Double-blind randomized trial to optimize steroid regimen in Duchenne MD (website)
  • Function of the Zinc Finger Protein ZPR1 in neurodegeneration (website)
  • Functional consequences of HSPB1 mutations that result in motor neuron disease (website)
  • Functional Dissection of Huntington’s Disease Protein Huntingtin Using Drosophila (website)
  • Functional dissection of medullary respiratory microcircuits (website)
  • Functional role of Ascl1 in the developing spinal cord (website)
  • GABA-A Receptor Alpha 1 Subunit Mutations and Epilepsy (website)
  • Gene delivery of MG53 for muscle membrane repair and functional improvement in a (website)
  • Gene therapy for alpha-mannosidosis (website)
  • Gene Therapy for Batten Disease Using AAVrh.10 Expressing Human CLN2 cDNA (website)
  • Genetic Dissection of Glioblastoma: Cell of Origin (website)
  • Genetic Mechanisms of Motor Neuron Degeneration (website)
  • Genetic modifiers of motor neuron degeneration (website)
  • Genetics and Molecular Biology of Parkinsonism (website)
  • Genetics of Familial Epilepsy Syndromes (website)
  • Glycolipid Replacement Therapy for Huntington’s Disease (website)
  • High-speed simulation of developmental motor disorders (website)
  • HSF1 as a therapeutic target in neurodegenerative disease (website)
  • Huntington’s Disease Antisense Transcript (website)
  • Identification and characterization of RNA-protein interactions in SCA3-disease (website)
  • Identification of glioma tumor-initiating cells (website)
  • Identifying Epileptogenic Tubers in TSC (website)
  • Identifying the Nmnat Axon Protection Pathway Via Multiple Screening Paradigms (website)
  • In vivo modeling of TDP-43 toxicity (website)
  • Inhibiting the Androgen Receptor N/C Interaction to Treat SBMA (website)
  • Investigating a novel form of Charcot-Marie-Tooth disease (website)
  • Investigation of post-translational modifications in WT SOD1 in sporadic ALS (website)
  • Ion Channel Modulation by The Prion Protein: A Novel Toxic Mechanism (website)
  • Iron in the pathogenesis of Friedreich’s ataxia (website)
  • Joubert syndrome and related disorders of hindbrain development (website)
  • Local Circuit Properties of Mouse Corticospinal Neurons (website)
  • M1 and M4 mAChRs in striatum-implication in treatment of movement disorders (website)
  • Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy (website)
  • Manipulating temporal and spacial CaMKII activity in Angelman Syndrome (website)
  • Mechanism of LTBP4 in muscular dystrophy (website)
  • Mechanisms governing Nuclear Factor I gene induction and function during the ini (website)
  • Mechanisms of Glioma Resistance to EGFR Inhibition (website)
  • Mechanisms of injury and acute repair of axons in TBI (website)
  • MeCP2 Modulation of BDNF Signaling: Shared Mechanisms of Rett and Autism (website)
  • Mitochondria therapeutic in Huntington’s disease (website)
  • Molecular and cellular mechanism of Microcephaly (website)
  • Molecular Targeting of L1CAM to Inhibit Glioblastoma Stem Cell Invasion (website)
  • Monoallelic repair of expanded huntingtin by trans-splicing (website)
  • Motor Neuron Disease in Mouse Models of ALS: Where Does the End Begin (website)
  • Neural dysfunction and neuroinflammation in African brain disorders (website)
  • Neural Stem Cell Based Virotherapy for Malignant Glioma (website)
  • Neurodegenerative Diseases: A New Class of Primary Developmental Disorders? (website)
  • Neurofibromin Regulation Of Neural Stem Cell Function In Vitro And In Vivo (website)
  • Neuroimmunology of Vaccines in Adoptive T-cell Therapy for Brain Tumor (website)
  • Neuropathology of severe malaria in Thailand: MRI studies (website)
  • Next Generation gene discovery in neurogenetics (website)
  • Noninvasive Assessment of Neuromuscular Disease using Electrical Impedance (website)
  • Non-redundant functions of Hu proteins as neuron-specific splicing regulators (website)
  • Novel Histone Deacetylase Inhibitors as Therapeutics for Huntington’s Disease (website)
  • Novel Hsp90 Inhibitors: Proof-of-Principle for Treatment of Motor Neuron Diseases (website)
  • NT-3 gene therapy to improve peripheral nerve function induced by genetic defect (website)
  • Optimizing EGFR Targeted Therapy in Malignant Glioma (website)
  • Oxidation-dependent mutant huntingtin oligomers and Huntington’s disease pathogen (website)
  • Pathogenic Mechanisms in Fragile X Tremor Ataxia Syndrome (website)
  • Pathogenic Mechanisms of ALS-Linked TDP Gene Mutation (website)
  • Pathophysiology and Gene Replacement Strategies for Arginase Deficiency (website)
  • PGC-1alpha and GABAergic Dysfunction in Huntington Disease (website)
  • Pre-Clinical and Clinical Evaluation of Skeletal Muscle Activator, CK-2017357 for (website)
  • Preclinical Development of Lead Compounds Targeting Myotonic Dystrophy (website)
  • Pre-clinical Translation of Regulatory T-cell Inhibition in Brain Tumors (website)
  • Precursor cell-niche interactions and the genesis of brainstem gliomas (website)
  • Preventing Glioma Cancer Stem Cell Dispersal Using Cdc2 Kinase Inhibitors (website)
  • Prospective Study of Clinically Non-functioning Pituitary Adenomas (website)
  • Protein Acetylation and Selective Autophagy (website)
  • Proteolytic Fragments of Mutant Huntingtin Protein in HD Brain Regions (website)
  • Regulated Splicing of the Cholinergic Gene Locus (website)
  • Regulation of SMN and Identification of its Downstream Target (website)
  • Regulation of the Na/K Pump by RNA Editing (website)
  • Regulation, signaling, and dynamics of glucan phosphatases (website)
  • Retrograde Motility of Mitochondria (website)
  • RNA interference as therapy for spinocerebellar ataxia type 3 (website)
  • RNAi therapy for Huntingtons disease: safety &efficacy in the nonhuman primate (website)
  • RNA-induced transcriptional gene silencing in Friedreich ataxia (website)
  • Role of ASK1 for GAPDH-Siah binding and GAPDH-Siah stress-signaling in HD (website)
  • Role of Cofilin and Wnt Signaling in Neural Crest Development (website)
  • Role of CUGBP1 in skeletal muscle wasting in myotonic dystrophy (website)
  • Role of MELK in glioblastomas (website)
  • Role of NKCC1 on Brain Tumor Stem Cell Migration After EGF and Slit-2 Stimulation (website)
  • Role of Parkin in Familial and Idiopathic Parkinson’s Disease (website)
  • Role of Rab-5 regulators in neuronal development (website)
  • Role of RIG-like receptors in virally induced CNS inflammation (website)
  • Role of TEMs in brain tumor dispersal (website)
  • Role of the polarity RhoGEF PLEKHG5 on brain tumor dispersal (website)
  • Signaling Mechanisms of the Dystrophin-Glycoprotein Complex (website)
  • Simultaneous Analysis of Neurochemical and Behavioral/Cognitive Events In Vivo (website)
  • Single chain Fragments of variable regions in the treatment of Familial ALS (website)
  • Skeletal Muscle Mechanisms of Disease in ALS (website)
  • Small Molecule Neurotrophin Mimetics to Treat Huntington’s Disease (website)
  • SMN Control of snRNP Biogenesis: Role in RNA Splicing and Motor Neuron Survival (website)
  • Somatic mutation detection in brain AVM by massively high-throughput sequencing (website)
  • Spinal Muscular Atrophy: Cell-based drug screens for treatment of axonal defects (website)
  • Stabilizing ALS SOD1 Variants by Crosslinking Subunits (website)
  • Structure, Folding, and Misfolding of PMP22 (website)
  • Substrate Optimization Therapy: A Novel Therapy for Mucopolysaccharidosis (website)
  • Targeted Delivery of S100B Inhibitory Peptide to SCA1 Mouse Cerebellum (website)
  • Targeted screening for Taenia solium tapeworms (website)
  • Targeted Treatments for Invasive CNS Tumors (website)
  • Targeting physiologic changes as a route towards therapy for degenerative ataxias (website)
  • Targeting SMN2 Alternative Splicing for the Treatment of Spinal Muscular Atrophy (website)
  • Targeting the PI3K/Akt pathway in High Grade Glioma (website)
  • TDP-43 and FUS RNA pathways in motor neuron degeneration (website)
  • TDP-43 Proteinopathies in ALS-Dementia (website)
  • The Anaphase Promoting Complex and Cell Cycle Exit (website)
  • The function of ATM protein in the biology of the adult neuron CNS: (website)
  • The Pathogenesis of Facioscapulohumeral Muscular Dystrophy (website)
  • The PPAR-delta pathway in neural function and Hungtington’s disease neuropatholog (website)
  • The Role of a Cerebello-Thalamo-Basal Ganglia Pathway in Dystonia (website)
  • The Role of Astrocytes in Huntington’s Disease (website)
  • The role of cortical neuron and circuit function in tau induced neurodegeneration (website)
  • The Role of PRAS40 in Integrating Cell Growth and Stress Response Signaling (website)
  • The role of RPTPzeta in models of Congenital Muscular Dystrophies (website)
  • The Role of Sam68 in Synaptic Transmission and Plasticity (website)
  • The Role of Stress Granules in the Pathophysiology of TDP-43 (website)
  • The Role of THAP1 in Dystonia (website)
  • The Role of White Matter Progenitors in Glioma Formation and Progression (website)
  • Therapeutic Targeting of Abnormal Conformation in Neurodegenerative Disease (website)
  • Theraupeutic Candidates for Huntington’s Disease: From Lead to IND (website)
  • Toward Multi-Center MR Brain Perfusion (website)
  • Towards Gene Therapy for Angelman Syndrome (website)
  • Training in Neurogenetics: Function of NUP107 in Human and Mouse (website)
  • Transgenic Study of ALS-Linked CCS Mutations (website)
  • Translational Development of Replication-Competent Retrovirus Vectors (website)
  • Understanding and enhancing mechanisms of priming in cancer immunotherapy (website)
  • Using CD4+ T cells as a candidate therapy to slow disease progression in ALS (website)
  • XIAP Gene Therapy in Huntington’s Disease (website)