Reduced C9orf72 expression exacerbates polyGR toxicity in patient iPSC-derived motor neurons and a Type I protein arginine methyltransferase inhibitor reduces that toxicity

INTRODUCTION: Intronic repeat expansions in the C9orf72 gene are the most frequent known single genetic causes of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). These repeat expansions are believed to result in both loss-of-function and toxic gain-of-function. Gain-of-functio...

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Detalles Bibliográficos
Autores principales: Dane, Therese L., Gill, Anna L., Vieira, Fernando G., Denton, Kyle R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10149854/
https://www.ncbi.nlm.nih.gov/pubmed/37138766
http://dx.doi.org/10.3389/fncel.2023.1134090

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10149854/
https://www.ncbi.nlm.nih.gov/pubmed/37138766
http://dx.doi.org/10.3389/fncel.2023.1134090

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