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Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders

4 Nov 2019

Abstract excerpt

Polyglutamine (polyQ) disorders are a group of nine neurodegenerative diseases that share a common genetic cause, which is an expansion of CAG repeats in the coding region of the causative genes that are otherwise unrelated. The trinucleotide expansion encodes for an expanded polyQ tract in the respective proteins, resulting in toxic gain-of-function and eventually in neurodegeneration. Currently, no...

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PMID: 31738395
DOI: 10.1093/brain/awz328

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