心血管疾病的位点特异性基因治疗。
Site-specific gene therapy for cardiovascular disease.
作者信息
Fishbein Ilia, Chorny Michael, Levy Robert J
机构信息
The Children's Hospital of Philadelphia, Abramson Research Center, Philadelphia, PA 19104, USA.
出版信息
Curr Opin Drug Discov Devel. 2010 Mar;13(2):203-13.
Abstract
Gene therapy holds considerable promise for the treatment of cardiovascular disease and may provide novel therapeutic solutions for both genetic disorders and acquired pathophysiologies such as arteriosclerosis, heart failure and arrhythmias. Recombinant DNA technology and the sequencing of the human genome have made a plethora of candidate therapeutic genes available for cardiovascular diseases. However, progress in the field of gene therapy for cardiovascular disease has been modest; one of the key reasons for this limited progress is the lack of gene delivery systems for localizing gene therapy to specific sites to optimize transgene expression and efficacy. This review summarizes progress made toward the site-specific delivery of cardiovascular gene therapy and highlights selected promising novel approaches.
摘要
基因治疗在心血管疾病的治疗方面具有巨大潜力，并且可能为遗传性疾病以及诸如动脉硬化、心力衰竭和心律失常等后天性病理生理状况提供新的治疗方案。重组DNA技术和人类基因组测序已催生出大量可用于治疗心血管疾病的候选治疗基因。然而，心血管疾病基因治疗领域的进展较为有限；取得这一有限进展的关键原因之一是缺乏能够将基因治疗定位到特定部位以优化转基因表达和疗效的基因递送系统。本综述总结了心血管疾病基因治疗在定点递送方面取得的进展，并重点介绍了一些有前景的新方法。
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本文引用的文献
1
Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines.经皮经心内膜递送自我互补腺相关病毒6实现犬类心脏整体基因转移
Mol Ther. 2008 Dec;16(12):1953-1959. doi: 10.1038/mt.2008.202. Epub 2016 Dec 8.
2
Stereotaxis Niobe magnetic navigation system for endocardial catheter ablation and gastrointestinal capsule endoscopy.用于心内膜导管消融和胃肠胶囊内镜检查的立体定向Niobe磁导航系统。
Expert Rev Med Devices. 2009 Sep;6(5):487-98. doi: 10.1586/erd.09.32.
3
Magnetically responsive biodegradable nanoparticles enhance adenoviral gene transfer in cultured smooth muscle and endothelial cells.磁性响应性可生物降解纳米颗粒增强培养的平滑肌细胞和内皮细胞中的腺病毒基因转导。
Mol Pharm. 2009 Sep-Oct;6(5):1380-7. doi: 10.1021/mp900017m.
4
Nitric oxide synthase gene therapy: progress and prospects.
Expert Opin Biol Ther. 2009 Jul;9(7):867-78. doi: 10.1517/14712590903002047.
5
Cell-controlled and spatially arrayed gene delivery from fibrin hydrogels.来自纤维蛋白水凝胶的细胞控制和空间排列的基因递送。
Biomaterials. 2009 Aug;30(22):3790-9. doi: 10.1016/j.biomaterials.2009.03.049. Epub 2009 Apr 23.
6
Drug releasing systems in cardiovascular tissue engineering.心血管组织工程中的药物释放系统。
J Cell Mol Med. 2009 Mar;13(3):422-39. doi: 10.1111/j.1582-4934.2008.00532.x.
7
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