用于中枢神经系统基因治疗的病毒载体和递送策略。
Viral vectors and delivery strategies for CNS gene therapy.
作者信息
Gray Steven J, Woodard Kenton T, Samulski R Jude
机构信息
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
出版信息
Ther Deliv. 2010 Oct;1(4):517-34. doi: 10.4155/tde.10.50.
Abstract
This review aims to provide a broad overview of the targets, challenges and potential for gene therapy in the CNS, citing specific examples. There are a broad range of therapeutic targets, with very different requirements for a suitable viral vector. By utilizing different vector tropisms, novel routes of administration and engineered promoter control, transgenes can be targeted to specific therapeutic applications. Viral vectors have proven efficacious in preclinical models for several disease applications, spurring several clinical trials. While the field has pushed the limits of existing adeno-associated virus-based vectors, a next generation of vectors based on rational engineering of viral capsids should expand the application of gene therapy to be more effective in specific therapeutic applications.
摘要
本综述旨在通过引用具体实例，对中枢神经系统基因治疗的靶点、挑战及潜力进行广泛概述。治疗靶点范围广泛，对合适的病毒载体有非常不同的要求。通过利用不同的载体嗜性、新的给药途径和工程化启动子控制，转基因可靶向特定的治疗应用。病毒载体已在多种疾病应用的临床前模型中证明有效，从而推动了多项临床试验。虽然该领域已突破了现有腺相关病毒载体的极限，但基于病毒衣壳合理工程设计的新一代载体应能扩大基因治疗的应用范围，使其在特定治疗应用中更有效。
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