Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Nat Rev Drug Discov. 2019 May;18(5):358-378. doi: 10.1038/s41573-019-0012-9.
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. Continued study of AAV biology and increased understanding of the associated therapeutic challenges and limitations will build the foundation for future clinical success.
腺相关病毒 (AAV) 载体是治疗多种人类疾病的基因传递的主要平台。在开发临床上理想的 AAV 衣壳、优化基因组设计和利用革命性生物技术方面的最新进展，为基因治疗领域的发展做出了巨大贡献。在 AAV 介导的基因替换、基因沉默和基因编辑的临床前和临床成功，帮助 AAV 作为理想的治疗载体获得了广泛认可，有两种基于 AAV 的治疗药物在欧洲或美国获得了监管批准。对 AAV 生物学的持续研究和对相关治疗挑战和限制的深入了解，将为未来的临床成功奠定基础。
