用于治疗性基因编辑的CRISPR/Cas9体内递送：进展与挑战
In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges.
作者信息
Mout Rubul, Ray Moumita, Lee Yi-Wei, Scaletti Federica, Rotello Vincent M
机构信息
Department of Chemistry, University of Massachusetts , 710 North Pleasant Street, Amherst, Massachusetts 01003, United States.
出版信息
Bioconjug Chem. 2017 Apr 19;28(4):880-884. doi: 10.1021/acs.bioconjchem.7b00057. Epub 2017 Mar 17.
Abstract
The successful use of clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the CRISPR components. There are, however, major challenges on the delivery front. In this Topical Review, we will highlight recent developments in CRISPR delivery, and we will present hurdles that still need to be overcome to achieve effective in vivo editing.
摘要
将基于成簇规律间隔短回文重复序列（CRISPR）/Cas9的基因编辑成功用于治疗需要在体内高效递送CRISPR组件。然而，在递送方面存在重大挑战。在本专题综述中，我们将重点介绍CRISPR递送的最新进展，并阐述为实现有效的体内编辑仍需克服的障碍。
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