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基因治疗的进展与前景:通过全身给药进行的非病毒基因治疗

Gene therapy progress and prospects: non-viral gene therapy by systemic delivery.

作者信息

Li S-D, Huang L

机构信息

Division of Molecular Pharmaceutics, School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599-7360, USA.

出版信息

Gene Ther. 2006 Sep;13(18):1313-9. doi: 10.1038/sj.gt.3302838.

Abstract

Non-viral vectors continue to be an attractive alternative to viral vectors due to their safety, versatility and ease of preparation and scale-up. Over the past few years, investigators have been successful in developing gene carriers that can be targeted to the disease site. Several different delivery vectors for systemic use have been developed by different groups for plasmid DNA and oligonucleotide. Most of them are designed for targeted tumor therapy. The mechanism of inflammatory toxicity, the major toxicity of cationic lipoplex, has been studied and managed. In this review, we focus on the progress made over the last 2 years. We also discuss some future prospects for gene delivery.

摘要

由于非病毒载体具有安全性、多功能性以及易于制备和扩大规模等特点,它们仍然是病毒载体颇具吸引力的替代选择。在过去几年里,研究人员已成功开发出可靶向疾病部位的基因载体。不同团队针对质粒DNA和寡核苷酸开发了几种不同的全身用递送载体。其中大多数是为靶向肿瘤治疗而设计的。阳离子脂质体复合物的主要毒性——炎症毒性的机制已得到研究和控制。在本综述中,我们重点关注过去两年所取得的进展。我们还讨论了基因递送的一些未来前景。

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