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核酶的可控、靶向性细胞内表达:进展与问题

Controlled, targeted, intracellular expression of ribozymes: progress and problems.

作者信息

Rossi J J

机构信息

Center for Molecular Biology and Gene Therapy, Loma Linda University School of Medicine, CA 92350, USA.

出版信息

Trends Biotechnol. 1995 Aug;13(8):301-6. doi: 10.1016/S0167-7799(00)88969-6.

Abstract

Significant progress has been made in the development of ribozymes for a wide variety of intracellular applications, including human gene therapy. Further advances are likely to come from innovative strategies to improve the delivery, expression, co-localization, targeting specificity and substrate turnover of ribozymes. In order to stimulate problem solving in these areas, this article discusses examples of recent successes in intracellular ribozyme applications, and identifies some of the obstacles that remain. In addition, some testable, but as yet untried, ideas for overcoming several of these obstacles are presented.

摘要

在用于包括人类基因治疗在内的各种细胞内应用的核酶开发方面已经取得了重大进展。进一步的进展可能来自于创新策略,以改善核酶的递送、表达、共定位、靶向特异性和底物周转。为了激发这些领域的问题解决,本文讨论了细胞内核酶应用近期成功的例子,并指出了仍然存在的一些障碍。此外,还提出了一些可测试但尚未尝试的克服其中一些障碍的想法。

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