An Emerging Adeno-Associated Viral Vector Pipeline for Cardiac Gene Therapy

被引：30

作者：

Asokan, Aravind ^{[1
,2
]}

Samulski, R. Jude ^{[1
,3
]}

机构：

[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27516 USA

[2] Univ N Carolina, Dept Genet, Chapel Hill, NC 27516 USA

[3] Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27516 USA

来源：

HUMAN GENE THERAPY | 2013年 / 24卷 / 11期

基金：

美国国家卫生研究院;

关键词：

EXTRACELLULAR-SUPEROXIDE DISMUTASE; LONG-TERM EXPRESSION; HEART-FAILURE; VIRUS SEROTYPES; TRANSENDOCARDIAL DELIVERY; INTRACORONARY DELIVERY; SYSTEMIC TRANSDUCTION; RECEPTOR FOOTPRINT; CORONARY INFUSION; TRANSFER SUPERIOR;

D O I：

10.1089/hum.2013.2515

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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.

引用

页码：906 / 913

页数：8

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