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Letter to the Editor
Cell Research 27, 440-443 (March 2017) | doi:10.1038/cr.2017.16
A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo
Chao Jiang1, *, Miao Mei1, *, Bin Li2, *, Xiurui Zhu3, *, Wenhong Zu1, Yujie Tian1, Qiannan Wang1, Yong Guo3, Yizhou Dong2, Xu Tan1
1School of Pharmaceutical Sciences, Center for Infectious Disease Re-
search, 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
*These four authors contributed equally to this work.
Correspondence: Xu Tan a, Yizhou Dong b
a Email: [email protected].
b Email: [email protected]
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.
"Here we show that by using an optimized formula of our newly developed
lipid-like nanoparticles (LLNs) [4], we are able to effectively deliver Cas9 mRNA and single-guide RNA (sgRNA) to the liver and achieve in vivo targeting of
HBV DNA and the proprotein convertase subtilisin/kexin type 9 (pcsk9) gene, a therapeutic target for treating hypercholesterolemia [5]. To the best of our
knowledge, this is the first report on non-viral delivery of CRISPR/Cas9 system components (mRNA + sgR-NA) in adult animals. "
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发表于 2017-3-4 21:05
