Epigenetic Small Molecules Rescue Nucleocytoplasmic Transport and DNA Damage Phenotypes in C9ORF72 ALS/FTD

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disease with available treatments only marginally slowing progression or improving survival. A hexanucleotide repeat expansion mutation in the <i>C9ORF72</i> gene is the most commonly known genetic cause of...

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Autores principales: Melina Ramic, Nadja S. Andrade, Matthew J. Rybin, Rustam Esanov, Claes Wahlestedt, Michael Benatar, Zane Zeier
Formato: article
Lenguaje:EN
Publicado: MDPI AG 2021
Materias:
ALS
PR
GR
Acceso en línea:https://doaj.org/article/33eb8aaf3f164723a92feba8d8aaab4a
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