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)