期刊
HUMAN GENE THERAPY
卷 24, 期 4, 页码 375-392出版社
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2012.241
关键词
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资金
- National Heart, Lung and Blood Institute [NIH 1-R01-HL083078-01A2]
- Gene Therapy Resource Program of the National Heart, Lung and Blood Institute [NIH P30-DK047757]
Heart diseases are major causes of morbidity and mortality in Western society. Gene therapy approaches are becoming promising therapeutic modalities to improve underlying molecular processes affecting failing cardiomyocytes. Numerous cardiac clinical gene therapy trials have yet to demonstrate strong positive results and advantages over current pharmacotherapy. The success of gene therapy depends largely on the creation of a reliable and efficient delivery method. The establishment of such a system is determined by its ability to overcome the existing biological barriers, including cellular uptake and intracellular trafficking as well as modulation of cellular permeability. In this article, we describe a variety of physical and mechanical methods, based on the transient disruption of the cell membrane, which are applied in nonviral gene transfer. In addition, we focus on the use of different physiological techniques and devices and pharmacological agents to enhance endothelial permeability. Development of these methods will undoubtedly help solve major problems facing gene therapy.
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