Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG

The clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 system is a research hotspot in gene therapy. However, the widely used Streptococcus pyogenes Cas9 (WT-SpCas9) requires an NGG protospacer adjacent motif (PAM) for target recognition, thereby restricting targetable disease mutatio...

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Udgivet i:Commun Biol
Main Authors: Oura, Seiya, Noda, Taichi, Morimura, Naoko, Hitoshi, Seiji, Nishimasu, Hiroshi, Nagai, Yoshitaka, Nureki, Osamu, Ikawa, Masahito
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8222283/
https://ncbi.nlm.nih.gov/pubmed/34163001
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s42003-021-02304-w
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