Article

Safety and efficacy of <i>C9ORF72</i> -repeat RNA nuclear export inhibition in amyotrophic lateral sclerosis

2021-04-12

Abstract excerpt

<h4>Background</h4> Loss of motor neurons in amyotrophic lateral sclerosis (ALS) leads to progressive paralysis and death. Dysregulation of thousands of RNA molecules with roles in multiple cellular pathways hinders the identification of ALS-causing alterations over downstream changes secondary to the neurodegenerative process. How many and which of these pathological gene expression changes require therapeutic n...

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Literature Corpus work: ee2c9565-c65f-5e7f-bd2f-d692abae6ef9
DOI: 10.1101/2021.04.12.438950

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