An Emerging Adeno-Associated Viral Vector Pipeline for Cardiac Gene Therapy
An Emerging Adeno-Associated Viral Vector Pipeline for Cardiac Gene Therapy
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DOI：
10.1089/hum.2013.2515
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发表时间：
2013-11-01
影响因子：
4.2
通讯作者：
Samulski, R. Jude
中科院分区：
文献类型：
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作者：
Asokan, Aravind;Samulski, R. Jude
自然产生的腺相关病毒（AAV）分离株在不同宿主中表现出不同的组织趋向性。某些AAV毒株的心脏转导尤其强劲。这些AAV毒株在临床前和临床环境中的成功应用，重点是治疗心血管疾病的报道仍在继续。与此同时，这些研究也突出了AAV趋向性、转导效率和免疫力等跨物种差异等挑战。我们对AAV衣壳结构和生物学的理解不断进步，为设计可能解决这些问题的改进载体提供了理论依据。本报告概述了嗜心性AAV，现有的知识差距，以及心脏基因治疗临床可行的新工程AAV菌株。
The naturally occurring adeno-associated virus (AAV) isolates display diverse tissue tropisms in different hosts. Robust cardiac transduction in particular has been reported for certain AAV strains. Successful applications of these AAV strains in preclinical and clinical settings with a focus on treating cardiovascular disease continue to be reported. At the same time, these studies have highlighted challenges such as cross-species variability in AAV tropism, transduction efficiency, and immunity. Continued progress in our understanding of AAV capsid structure and biology has provided the rationale for designing improved vectors that can possibly address these concerns. The current report provides an overview of cardiotropic AAV, existing gaps in our knowledge, and newly engineered AAV strains that are viable candidates for the cardiac gene therapy clinic.