EFFICACY AND SAFETY EVALUATION OF A LEAD GENE THERAPY CANDIDATE IN PROPIONIC ACIDEMIA (PA) DISEASE MOUSE MODELS AND BIOANALYTICAL ASSAY METHODS DEVELOPMENT.

国家转化科学推进中心（NCATS）通过其罕见和被忽视疾病治疗（TRND）计划，正在与国家人类基因组研究所（NHGRI）合作，推进AAV9丙酰辅酶A羧化酶（PCCA）基因治疗候选药物，作为丙酸血症（PA）的可能治疗方法。该任务订单的总体目标是让承包商评估腺相关病毒9（AAV9）丙酰辅酶A羧化酶（PCCA）基因治疗候选物在丙酸血症（PA）疾病小鼠模型中的有效性和安全性，该模型先前在NHGRI开发，并开发生物分析测定方法，用于测量PCCA转基因表达产生的生物分布和酶活性。

The National Center for Advancing Translational Sciences (NCATS), through its Therapeutics for Rare and Neglected Diseases (TRND) program, is collaborating with the National Human Genome Research Institute (NHGRI) to advance an AAV9 Propionyl-CoA Carboxylase (PCCA) gene therapy candidate as a possible treatment for Propionic Acidemia (PA). The overall objective of this task order is for the contractor to evaluate the efficacy and safety of an Adeno-associated virus 9 (AAV9) Propionyl-CoA Carboxylase (PCCA) gene therapy candidate in propionic acidemia (PA) disease mouse models, previously developed at NHGRI, and develop bioanalytical assay methods that will be used to measure biodistribution and enzyme activity resulting from PCCA transgene expression.