BET bromodomain inhibitors PFI-1 and JQ1 are identified in an epigenetic compound screen to enhance C9ORF72 gene expression and shown to ameliorate C9ORF72-associated pathological and behavioral abnormalities in a C9ALS/FTD model

BACKGROUND: An intronic GGGGCC (G4C2) hexanucleotide repeat expansion (HRE) in the C9ORF72 gene is the most common cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), referred to as C9ALS/FTD. No cure or effective treatment exist for C9ALS/FTD. Three major molecular mecha...

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Detalles Bibliográficos
Autores principales: Quezada, Esteban, Cappelli, Claudio, Diaz, Iván, Jury, Nur, Wightman, Nicholas, Brown, Robert H., Montecino, Martín, van Zundert, Brigitte
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7962347/
https://www.ncbi.nlm.nih.gov/pubmed/33726839
http://dx.doi.org/10.1186/s13148-021-01039-z

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