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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Fused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). To explore the mechanisms by which mutant FUS causes neurodegeneration in ALS-FTD, we generated...

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Detalles Bibliográficos
Autores principales: Korobeynikov, Vladislav A., Lyashchenko, Alexander K., Blanco-Redondo, Beatriz, Jafar-Nejad, Paymaan, Shneider, Neil A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8799464/
https://www.ncbi.nlm.nih.gov/pubmed/35075293
http://dx.doi.org/10.1038/s41591-021-01615-z