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心脏疾病的基因和细胞治疗传递。

Delivery of gene and cellular therapies for heart disease.

机构信息

Heart Failure Research Group, Baker IDI Heart and Diabetes Institute, P.O. Box 6492, St. Kilda Rd Central, Melbourne, Victoria, 8008, Australia.

出版信息

J Cardiovasc Transl Res. 2010 Aug;3(4):417-26. doi: 10.1007/s12265-010-9190-x. Epub 2010 May 11.

Abstract

Although there has been considerable interest in the utilization of gene and cellular therapy for heart disease in recent years, there remain critical questions prior to widespread promotion of therapy, and key among these issues is the delivery method used for both gene therapy and cellular therapy. Much of the failure of gene and cellular therapy can be explained by the biological therapy itself; however, certainly there is a critical role played by the delivery technique, in particular, those that have been adapted from routine clinical use such as intravenous and intracoronary injection. Development of novel techniques to deliver gene and cellular therapy has ensued with some preclinical and even clinical success, though questions regarding safety, invasiveness, and repeatability remain. Here, we review techniques for gene and cellular therapy delivery, both existing and adapted techniques, and novel techniques that have emerged recently at promoting improved efficacy of therapy without the cost of systemic distribution. We also highlight key issues that need to be addressed to improve the chances of success of delivery techniques to enhance therapeutic benefit.

摘要

尽管近年来人们对基因和细胞疗法在心脏病中的应用产生了浓厚的兴趣,但在广泛推广治疗方法之前,仍存在一些关键问题,其中最重要的是基因疗法和细胞疗法所使用的传递方法。基因和细胞疗法的大部分失败可以用其生物学治疗本身来解释;然而,传递技术确实起到了关键作用,特别是那些从常规临床应用中发展而来的技术,如静脉内和冠状动脉内注射。随着一些临床前甚至临床的成功,新型技术已经随之出现,用于传递基因和细胞疗法,尽管仍存在安全性、侵袭性和可重复性等问题。在这里,我们综述了基因和细胞疗法传递的技术,包括现有的和改进的技术,以及最近出现的新技术,这些技术在提高治疗效果的同时,避免了全身分布的成本。我们还强调了需要解决的关键问题,以提高传递技术的成功率,从而提高治疗效果。

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