Optimizing gene delivery vectors for the treatment of heart disease

被引：4
作者：
Gray, Steven J. [1 ]
Samulski, R. Jude [1 ]
机构：
[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
来源：
EXPERT OPINION ON BIOLOGICAL THERAPY | 2008年 / 8卷 / 07期
关键词：
AAV; Ad; adenovirus; adeno-associated virus; gene therapy; heart; lentivirus; myocardium; plasmid;
D O I：
10.1517/14712598.8.7.911
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：
Background: Cardiac gene therapy is approaching reality, with clinical trials entering Phase II/III. Even so, challenges exist to improve the efficacy of even the most successful therapies. Objective: The merits of different gene therapy vectors are weighed to assess the current feasibility of each in specific cardiac applications. Major obstacles are discussed, along with recent advances in vector development to overcome or circumvent those difficulties. Methods: This review focuses primarily on gene delivery via naked DNA, adenovirus, lentivirus, and adeno-associated virus (AAV) vectors. Conclusion: Gene therapy via adenovirus and AAV vectors has developed into a promising option for the treatment of heart disease. The merits of gene therapy compared with emerging stem cell and microRNA-based treatments are discussed.
引用
收藏
页码：911 / 922
页数：12
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