Volume 27, No 3, Mar 2017

ISSN: 1001-0602 
EISSN: 1748-7838 2018 
impact factor 17.848* 
(Clarivate Analytics, 2019)

Volume 27 Issue 3, March 2017: 440-443

LETTERS TO THE EDITOR

A non-viral CRISPR/Cas9 delivery system for therapeutic gene targeting in vivo

Chao Jiang^1,*, Miao Mei^1,*, Bin Li^2,*, Xiurui Zhu^3,*, Wenhong Zu¹, Yujie Tian¹, Qiannan Wang¹, Yong Guo³, Yizhou Dong² and Xu Tan¹

¹School of Pharmaceutical Sciences, Center for Infectious Disease Research, School of Medicine, Tsinghua University, Tsinghua-Peking Center for Life Sciences, Beijing 100084, China
²Division of Pharmaceutics and Pharmaceutical Chemistry, College of Pharmacy, The Ohio State University, Columbus, OH 43210, USA
³Department of Biomedical Engineering, School of Medicine, Tsinghua University, Beijing 100084, China Correspondence: Xu Tan, Email: xutan@tsinghua.edu.cn; Yizhou Dong, Email:(dong.525@osu.edu)

The clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) system has revolutionized biomedical research and facilitated the development of new therapies based on genome editing1. A major roadblock to achieve the therapeutic potential of the CRISPR/Cas system is the lack of a safe and effectiving in vivo delivery method. Adeno-associated virus (AAV)-assisted delivery of the CRISPR/Cas9 system has shown gene targeting efficacy in vivo, however, the long persistence and immunogenicity of AAV in the host prevent the wide therapeutic application of AAV-based CRISPR/Cas9 delivery2.

10.1038/cr.2017.16

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