遗传增强
生物
衣壳
腺相关病毒
转基因
基因传递
向性
病毒载体
载体(分子生物学)
杜氏肌营养不良
病毒学
转导(生物物理学)
病毒
基因
遗传学
重组DNA
生物化学
作者
Chengwen Li,R. Jude Samulski
标识
DOI:10.1038/s41576-019-0205-4
摘要
Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic effects have been achieved for other rare diseases, including haemophilia and Duchenne muscular dystrophy. However, current research indicates that the genetic modification of AAV vectors may further facilitate the success of AAV gene therapy. Vector engineering can increase AAV transduction efficiency (by optimizing the transgene cassette), vector tropism (using capsid engineering) and the ability of the capsid and transgene to avoid the host immune response (by genetically modifying these components), as well as optimize the large-scale production of AAV.
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