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治疗中寡核苷酸和基因递送新策略设计的最新进展、前景与问题

Recent advances, prospects and problems in designing new strategies for oligonucleotide and gene delivery in therapy.

作者信息

Romano G, Claudio P P, Kaiser H E, Giordano A

机构信息

Kimmel Cancer Institute, Jefferson Medical College, Thomas Jefferson University, Philadelphia, PA 19107, USA.

出版信息

In Vivo. 1998 Jan-Feb;12(1):59-67.

PMID:9575427
Abstract

The field of gene therapy has been in rapid expansion since the first submissions of gene therapy trials in the early 1990s which provided encouraging results. Since then, many gene therapy protocols have been approved for phase I clinical trials for the treatment of inherited genetic diseases and cancer. The possibility of employing gene transfer technology to treat AIDS and neurologic diseases is currently under evaluation. Many gene delivery systems have been developed for in vivo studies and therapy. The efficiency of in vivo gene transfer, however, still needs to be optimized, even though significant advances have recently been achieved in improving gene delivery, gene regulation and avoidance of immune responses. This review provides a general outline focusing on the description of the most common gene delivery systems and on their current applications in therapeutics.

摘要

自20世纪90年代初首次提交基因治疗试验并取得令人鼓舞的结果以来,基因治疗领域一直在迅速扩展。从那时起,许多基因治疗方案已被批准用于治疗遗传性疾病和癌症的I期临床试验。目前正在评估采用基因转移技术治疗艾滋病和神经疾病的可能性。已经开发了许多基因递送系统用于体内研究和治疗。然而,尽管最近在改善基因递送、基因调控和避免免疫反应方面取得了重大进展,但体内基因转移的效率仍需优化。本综述提供了一个总体概述,重点描述了最常见的基因递送系统及其目前在治疗中的应用。

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