Hum Gene Ther：腺相关病毒靶向肝脏进行基因治疗

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腺相关病毒（AAV）载体介导的肝靶向基因疗法已经进入临床人体试验阶段，如用于治疗血友病等。最近，一篇发表在《Human Gene Therapy》上的综述文章介绍了新技术发展如何促进AAV基因疗法在治疗肝功紊乱方面的应用以及存在的挑战。

在这篇叫做“Adeno-Associated Virus Gene Therapy for Liver Disease”的文章中，来自剑桥Dimension Therapeutics公司的Lisa Kattenhorn等人对AAV靶向肝脏进行基因治疗的历史进程进行了总结。他们认为临床前及临床研究让我们更深入了解了机体对AAV基因治疗的免疫反应。此外，他们也对AAV基因治疗的未来发展趋势和存在的挑战进行了总结，包括如何反复使用AAV基因治疗、如何降低其致癌风险等。

“基于AAV的肝靶向基因疗法是治疗基因突变疾病的有效手段，包括血友病及先天代谢异常等，”马萨诸塞大学医学院教授Terence R Flotte博士说道。“这篇综述对这个领域的现状进行了很全面而深刻的总结。”

来源：生物谷

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Adeno-Associated Virus Gene Therapy for Liver Disease

Lisa M. Kattenhorn, Christopher H. Tipper, Lorelei Stoica, Deborah S. Geraghty, Teresa L. Wright, K. Reed Clark, and Samuel C. Wadsworth

The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as other liver disorders. This review focuses on liver-directed AAV gene therapy, including historic context, current challenges, and future developments.

http://online.liebertpub.com/doi/10.1089/hum.2016.160