EFFICACY AND SAFETY EVALUATION OF LEAD GENE THERAPY CANDIDATE IN NEUROMUSCULAR JUNCTION (NMJ) DISEASE MOUSE MODELS AND BIOANALYTICAL ASSAY METHODS DEVELOPMENT

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The National Center for Advancing Translational Sciences (NCATS), through its Therapeutics for Rare and Neglected Diseases (TRND) program, is collaborating with the National Institutes of Neurological Disorders and Stroke (NINDS) to advance Adeno-associated virus gene therapy candidates as possible treatments for Congenital Myasthenic Syndromes (CMS). The overall objective of this task order is for the contractor to evaluate the efficacy and safety of AAV transgene gene therapy candidates in neuromuscular junction (NMJ) disease mouse models, previously developed at NINDS, and bioanalytical assay methods development to measure biodistribution and electrophysiology endpoints resulting from transgene expression. Efficacy and Safety Evaluation of gene therapy candidates in a Neuromuscular Junction (NMJ) disease mouse model and bioanalytical assay methods development.