Functional analysis of epilepsy-associated GABA receptor mutations using Caenorhabditis elegans.

Objective: GABA receptor subunit mutations pose a significant risk for genetic generalized epilepsy; however, there are over 150 identified variants, many with unknown or unvalidated pathogenicity. We aimed to develop in vivo models for testing GABA receptor variants using the model organism, Caenorhabditis elegans.

Methods: CRISPR-Cas9 gene editing was used to create a complete deletion of unc-49, a C.

New Therapeutic Uses for Existing Drugs.

Eighty percent of drugs that enter human clinical testing are never approved for use. This means that for every five drugs that make it into the clinic, there are four that failed to show effectiveness for treating the disease or condition the drug was designed to treat.This high failure rate means there are many existing, partially developed therapeutic candidates with known pharmacology, formulation, and potential toxicity.