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

国家推进转化科学中心（NCATS）通过其罕见和被忽视疾病治疗（TRND）计划，正在与美国国立神经疾病和中风研究所（NINDS）合作，以推进腺相关病毒基因治疗候选物作为先天性肌无力综合征（CMS）的可能治疗方法。 该任务订单的总体目标是让承包商评估AAV转基因基因治疗候选物在神经肌肉接头（NMJ）疾病小鼠模型中的有效性和安全性，以及生物分析测定方法的开发，以测量转基因表达引起的生物分布和电生理学终点。 基因治疗候选物在神经肌肉接头（NMJ）疾病小鼠模型中的功效和安全性评价以及生物分析测定方法的开发。

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.