Adeno-associated virus vector-based gene therapy:clinical use and safety information
ZHANG Jing-ye
ZHUO Yang
YU Jia-ning
ZHOU Long-rui
CHEN Yi-fei
Abstract：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.
Keywords：in vivo gene therapy productadeno-associated virus vectoradverse reactionclinical trial
Publication Date：2024-12-31
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：6( 2562-2567 )