Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

A large G(4)C(2)-repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Neuronal degeneration associated with this expansion arises from a loss of C9orf72 protein, the accumulation of RNA foci, the expression of dipeptid...

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Detalles Bibliográficos
Autores principales: Liu, Yuanjing, Dodart, Jean-Cosme, Tran, Helene, Berkovitch, Shaunna, Braun, Maurine, Byrne, Michael, Durbin, Ann F., Hu, Xiao Shelley, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Kenneth, Ruschel, Jörg, Shelke, Juili, Yang, Hailin, Yin, Yuan, Donner, Amy, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7870851/
https://www.ncbi.nlm.nih.gov/pubmed/33558503
http://dx.doi.org/10.1038/s41467-021-21112-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7870851/
https://www.ncbi.nlm.nih.gov/pubmed/33558503
http://dx.doi.org/10.1038/s41467-021-21112-8

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