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[基因转移系统与基因治疗]

[Gene transfer systems and gene therapy].

作者信息

Yilmaz Engin

机构信息

Hacettepe Universitesi Tip Fakültesi, Tibbi Biyoloji Anabilim Dali, Ankara.

出版信息

Mikrobiyol Bul. 2003 Jan;37(1):89-98.

Abstract

One of the most important developments in medicine is gene therapy. But before it can be accepted to be used widely, certain technical problems should be overcome, especially the methods of gene delivery. In order to modify a specific cell type or tissue, the therapeutic gene must be efficiently delivered to the cell, in such a way that the gene can be expressed at the appropriate level and for a sufficient duration. There are currently availible two gene delivery vehicles. The first consists of viral vectors, which take advantage of a variety of naturally occurring viruses to shuttle the foreign cargo to be integrated permanently into the host's chromosome. The second is non-viral vectors, which rely on direct delivery of either naked DNA or mixture of genes with cationic lipids (liposome). There have been over 600 clinical trials of gene therapy protocols. The majority of clinical trials of gene therapy have been involved in the treatment of cancer and most of them have been at phase I clinical trials. Phase II stage of gene therapy had started of the end of 1996.

摘要

医学领域最重要的进展之一是基因治疗。但在其被广泛接受使用之前,某些技术问题需要克服,尤其是基因递送方法。为了修饰特定的细胞类型或组织,治疗性基因必须有效地递送至细胞,使基因能够在适当水平表达并持续足够长的时间。目前有两种基因递送载体。第一种由病毒载体组成,它利用多种天然存在的病毒将外来物质穿梭运输,以永久整合到宿主染色体中。第二种是非病毒载体,它依靠直接递送裸DNA或基因与阳离子脂质(脂质体)的混合物。已经有超过600项基因治疗方案的临床试验。基因治疗的大多数临床试验都涉及癌症治疗,其中大部分处于I期临床试验阶段。基因治疗的II期阶段于1996年底开始。

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