Adeno-associated virus (AAV) vector has emerged as the most widely used vector for in vivo gene therapy products due to their advantages such as low integration risk and sustained exogenous expression.To date,six AAV vector-based products have obtained marketing authorization in the United States and the European Union,solidifying their position as the most prevailing category in gene therapy.This review conducts a comprehensive analysis encompassing data from both clinical trial phases and post-marketing observations.It examines the key aspects such as indications and usage,administration and dosage,clinical pharmacology,clinical studies,and adverse reactions,aiming to provide reference information for the development of gene therapy products in the future.
关键词
体内基因治疗产品/腺相关病毒载体/不良反应/临床试验
Key words
in vivo gene therapy product/adeno-associated virus vector/adverse reaction/clinical trial
维普
万方数据