Article

CRISPR/Cas9-based silencing of the <i>ATXN1</i> gene in Spinocerebellar ataxia type 1 (SCA1) fibroblasts

2020-07-04

Abstract excerpt

Spinocerebellar Ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disorder caused by a gain-of-function protein with toxic activities, containing an expanded polyQ tract in the coding region. Actually, there are no treatments available to delay the onset, stop or slow down the progression of this pathology. Many approaches developed over the years involve the use of siRNAs and antisense oligonucleoti...

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Literature Corpus work: 1c26a650-00b7-5f75-82cd-b4181e3f6cec
DOI: 10.1101/2020.07.04.187559

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