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逆转录病毒载体介导的基因治疗的最新进展:让旧载体发挥新作用

Recent advances in retrovirus vector-mediated gene therapy: teaching an old vector new tricks.

作者信息

Weber E, Anderson W F, Kasahara N

机构信息

USC Institute for Genetic Medicine, Los Angeles, CA 90033, USA.

出版信息

Curr Opin Mol Ther. 2001 Oct;3(5):439-53.

Abstract

Oncoretrovirus-based vectors have been shown to be a safe and reliable vector system that can achieve permanent integration of delivered transgenes. Successful application of these vectors for gene therapy has proven difficult due to their relatively low transduction efficiency; however, cumulative improvements in methodology have recently yielded promising clinical results. Furthermore, significant improvements in basic retrovirus vector technology now promise to revitalize the field. This review focuses on these important recent developments in the field of retrovirus-mediated gene transfer technology and its application to human diseases.

摘要

基于致癌性逆转录病毒的载体已被证明是一种安全可靠的载体系统,能够实现所传递转基因的永久整合。由于其相对较低的转导效率,这些载体在基因治疗中的成功应用已被证明具有挑战性;然而,方法学上的累积改进最近已产生了有前景的临床结果。此外,基本逆转录病毒载体技术的重大改进现在有望振兴该领域。本综述重点关注逆转录病毒介导的基因转移技术领域这些重要的最新进展及其在人类疾病中的应用。

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