Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA.
1] Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA. [2] Department of Bioengineering, and the Department of Molecular and Cell Biology, University of California, Berkeley, California 94720-3220, USA.
Nat Rev Genet. 2014 Jul;15(7):445-51. doi: 10.1038/nrg3742. Epub 2014 May 20.
Clinical gene therapy has been increasingly successful owing both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.
临床基因治疗由于对人类疾病的分子认识不断提高和基因传递技术的逐步改进而越来越成功。在这些技术中，基于腺相关病毒（AAV）的递送载体已被证明是安全有效的，并且在最近的一个案例中，已获得监管部门的批准。尽管病毒载体特性的缺陷将使此类成功扩展到许多其他人类疾病具有挑战性，但越来越多的新型工程和改进 AAV 载体及其遗传货物的方法正在帮助克服这些障碍。
