Article

Gene Editing for <i>ATXN3</i> Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity

2025-02-15

Abstract excerpt

<h4>ABSTRACT</h4> Machado-Joseph disease (MJD) is an autosomal dominantly-inherited neurodegenerative disorder, caused by an over-repetition of the polyglutamine-codifying region in the ATXN3 gene. Strategies based on the suppression of the deleterious gene products have demonstrated promising results in pre-clinical studies. Nonetheless, these strategies do not target the root cause of the disease. In order to...

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Literature Corpus work: d0ef73c4-a25c-540c-b4d2-e711a47c2da3
DOI: 10.1101/2025.02.14.637261

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