Article

A cell-penetrant peptide blocking <i>C9ORF72</i> -repeat RNA nuclear export suppresses neurodegeneration

2021-05-23

Abstract excerpt

Hexanucleotide repeat expansions in C9ORF72 are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a spectrum of incurable debilitating neurodegenerative diseases. Here, we report a novel ALS/FTD drug concept with in vivo and in vitro therapeutic activity in preclinical models of C9ORF72-ALS/FTD. Our data demonstrate that supplementation or oral administratio...

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Literature Corpus work: 90ee13bc-5827-5a1c-a970-560f0f6269b9
DOI: 10.1101/2021.05.23.445325

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