Dual-gRNA approach with limited off-target effect corrects C9ORF72 repeat expansion in vivo

C9ORF72 GGGGCC repeat expansion is the most common genetic cause for amyotrophic lateral sclerosis and frontotemporal dementia, which generates abnormal DNA and RNA structures and produces toxic proteins. Recently, efficacy of CRISPR/Cas9-mediated editing has been proven in treatment of disease. How...

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Detalles Bibliográficos
Autores principales: Piao, Xuejiao, Meng, Dawei, Zhang, Xue, Song, Qiang, Lv, Hailong, Jia, Yichang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983752/
https://www.ncbi.nlm.nih.gov/pubmed/35383205
http://dx.doi.org/10.1038/s41598-022-07746-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983752/
https://www.ncbi.nlm.nih.gov/pubmed/35383205
http://dx.doi.org/10.1038/s41598-022-07746-8

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