October 29, 2014
In addition or as an alternative to gene therapy, chemical drugs could offer benefits to treat certain genetic disorders resulting from loss-of-function (LOF) or gain-of-function (GOF) mutations. In an article published in Drug Discovery Today, Sun et al. found that most chemical drug binding sites are located far from the gene mutation locus. Based on this assumption, the authors suggest that chemical drugs could target certain genetic disorders without the gene mutation interfering with the drug binding site.
Over a hundred drug target pairs were matched with drug indications and genetic disease traits by comparing drug targets registered in the Therapeutic Target Database and genes registered in OMIM. These drug targets were studied to assess the influence of genetic mutations on candidate chemical drugs. The authors indicate that most of the studied genetic mutations had little influence on the drug binding sites. They suggest therefore that more chemical drugs should be considered as candidates to treat genetic and rare diseases.