In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges

被引：175

作者：

Mout, Rubul ^{[1
]}

Ray, Moumita ^{[1
]}

Lee, Yi-Wei ^{[1
]}

Scaletti, Federica ^{[1
]}

Rotello, Vincent M. ^{[1
]}

机构：

[1] Univ Massachusetts, Dept Chem, 710 North Pleasant St, Amherst, MA 01003 USA

来源：

BIOCONJUGATE CHEMISTRY | 2017年 / 28卷 / 04期

关键词：

MUSCULAR-DYSTROPHY; MEDIATED DELIVERY; MOUSE MODEL; HUMAN-CELLS; GENOME; CAS9; RNA; CRISPR-CAS9; NUCLEASES; ENDONUCLEASE;

D O I：

10.1021/acs.bioconjchem.7b00057

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：880 / 884

页数：5

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