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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 both sporadi...

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Main Authors: Melina Ramic, Nadja S. Andrade, Matthew J. Rybin, Rustam Esanov, Claes Wahlestedt, Michael Benatar, Zane Zeier
Format: Artigo
Language:Inglês
Published: MDPI AG 2021-11-01
Series:Brain Sciences
Subjects:
ALS
C9ORF72
nucleocytoplasmic transport
RAN proteins
PR
GR
Online Access:https://www.mdpi.com/2076-3425/11/11/1543
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