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

Seiya Oura and Taichi Noda et al. overcome the challenge of gene editing in CAG repeats, such as those causing Huntington’s Disease, using their recently developed SpCas9-NG variant. They demonstrate that SpCas9-NG can precisely edit and contract the CAG repeat tracks in a Huntington’s Disease mouse...

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Autores principales: Seiya Oura, Taichi Noda, Naoko Morimura, Seiji Hitoshi, Hiroshi Nishimasu, Yoshitaka Nagai, Osamu Nureki, Masahito Ikawa
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/6b8b24cc62624b4fbbeec478f8cf925c
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