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基因组学与基因治疗。人造染色体即将成为现实。

Genomics and gene therapy. Artificial chromosomes coming to life.

作者信息

Willard H F

机构信息

Department of Genetics and Center for Human Genetics at Case Western Reserve University and the Research Institute of Universi Hospitals of Cleveland, Cleveland, OH 44106, USA.

出版信息

Science. 2000 Nov 17;290(5495):1308-9. doi: 10.1126/science.290.5495.1308.

Abstract

One of the biggest obstacles to gene therapy is the delivery of the therapeutic gene to the target tissue so that it is appropriately expressed. In his Perspective, Willard looks at the potential advantages of using a human artificial chromosome to maintain expression of a therapeutic gene and discusses some of the hurdles yet to be overcome before this gene delivery system can be tried out in the clinic.

摘要

基因治疗最大的障碍之一是将治疗性基因传递到靶组织,使其能够得到适当表达。在他的《观点》文章中,威拉德探讨了使用人类人工染色体来维持治疗性基因表达的潜在优势,并讨论了在这种基因传递系统能够在临床中试用之前仍需克服的一些障碍。

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