Article

Small molecule v-ATPase inhibitor Etidronate lowers levels of ALS protein ataxin-2

2021-12-21

Abstract excerpt

<h4>Summary</h4> Antisense oligonucleotide therapy targeting ATXN2 —a gene in which mutations cause neurodegenerative diseases spinocerebellar ataxia type 2 and amyotrophic lateral sclerosis—has entered clinical trials in humans. Additional methods to lower ataxin-2 levels would be beneficial not only in uncovering potentially cheaper or less invasive therapies, but also in gaining greater mechanistic insight in...

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Literature Corpus work: 71715a8d-a6df-5bd7-87a8-53419ea8bb3c
DOI: 10.1101/2021.12.20.473567

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