基因编辑技术及其在疾病治疗中的研究进展

doi:10.16288/j.yczz.19-102

遗传 ›› 2019, Vol. 41 ›› Issue (7): 582-598.doi: 10.16288/j.yczz.19-102

基因编辑技术及其在疾病治疗中的研究进展

牛煦然,尹树明,陈曦,邵婷婷,李大力()

上海市调控生物学重点实验室,华东师范大学生命科学学院,上海 200241

收稿日期:2019-04-16 修回日期:2019-05-29 出版日期:2019-07-20 发布日期:2019-07-02
作者简介:牛煦然,本科在读,专业方向：生物科学。E-mail: 574569231@qq.com
基金资助:
国家自然科学基金项目资助(81670470);国家自然科学基金项目资助(81873685)

Gene editing technology and its recent progress in disease therapy

Xuran Niu,Shuming Yin,Xi Chen,Tingting Shao,Dali Li()

Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China

Received:2019-04-16 Revised:2019-05-29 Published:2019-07-20 Online:2019-07-02
Supported by:
Supported by the National Natural Science Foundation of China(81670470);Supported by the National Natural Science Foundation of China(81873685)

摘要/Abstract

摘要：

基因编辑是一种基于人工核酸酶的遗传操作技术,能精确地对DNA或RNA进行高效改造。基因编辑除了在基础研究、生物育种和药物筛选等方面展现了巨大前景之外,在疾病治疗(特别是基因遗传病)领域的前景与进展尤为引人注目。本文在介绍基因编辑技术的发展及其在疾病治疗中不同策略的基础上,重点围绕遗传疾病的基因治疗研究,综述了基因编辑技术(包括单碱基编辑和表观调控等技术)在血液系统、肝脏、肌肉和神经系统等疾病治疗的研究进展,并对基因编辑治疗疾病的未来发展进行了展望。

关键词: 基因编辑, 基因治疗, 遗传病

Abstract:

Gene editing is a genetic manipulation technology which utilizes bacterial nucleases to accurately and efficiently modify DNA or RNA. Gene editing has broad applications in basic research, breeding, and drug screening, and it is gaining validity and applicability to the therapy of many diseases especially genetic-based disease. In this review, we summarize the development of gene editing technology, its different strategies and applications in the treatment of disease, and the research of gene editing therapy for genetic diseases (including base editor and epigenetic regulation) in the treatment of disorders and diseases of the blood system, liver, muscle and nervous system. Finally, we discuss the future development prospects of gene editing therapy.

Key words: gene editing, gene therapy, genetic diseases

牛煦然,尹树明,陈曦,邵婷婷,李大力. 基因编辑技术及其在疾病治疗中的研究进展[J]. 遗传, 2019, 41(7): 582-598.

Xuran Niu,Shuming Yin,Xi Chen,Tingting Shao,Dali Li. Gene editing technology and its recent progress in disease therapy[J]. Hereditas(Beijing), 2019, 41(7): 582-598.

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基因编辑技术及其在疾病治疗中的研究进展

Gene editing technology and its recent progress in disease therapy

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