CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro

A GGGGCC(24+) hexanucleotide repeat expansion (HRE) in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), fatal neurodegenerative diseases with no cure or approved treatments that substantially slow disease progression or exten...

Descripción completa

Detalles Bibliográficos
Autores principales: Meijboom, Katharina E., Abdallah, Abbas, Fordham, Nicholas P., Nagase, Hiroko, Rodriguez, Tomás, Kraus, Carolyn, Gendron, Tania F., Krishnan, Gopinath, Esanov, Rustam, Andrade, Nadja S., Rybin, Matthew J., Ramic, Melina, Stephens, Zachary D., Edraki, Alireza, Blackwood, Meghan T., Kahriman, Aydan, Henninger, Nils, Kocher, Jean-Pierre A., Benatar, Michael, Brodsky, Michael H., Petrucelli, Leonard, Gao, Fen-Biao, Sontheimer, Erik J., Brown, Robert H., Zeier, Zane, Mueller, Christian
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/PMC9587249/
https://www.ncbi.nlm.nih.gov/pubmed/36271076
http://dx.doi.org/10.1038/s41467-022-33332-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9587249/
https://www.ncbi.nlm.nih.gov/pubmed/36271076
http://dx.doi.org/10.1038/s41467-022-33332-7

Ejemplares similares