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CRISPR/Cas9 Mediated Gene Modification Ameliorates Abnormal Phenotypes in SCA3/MJD Disease Patient Derived Induced Pluripotent Stem Cells

2020-10-12

Abstract excerpt

<title>Abstract</title> <p><bold>Background：</bold>Spinocerebellar ataxia type 3/Machado-Joseph disease (SCA3/MJD) is a progressive autosomal dominant neurodegenerative disease caused by abnormal CAG repeats in exon 10 of <italic>ATXN3</italic>. The accumulation of the mutant ataxin3 proteins carrying polyglutamine (polyQ) lead to selective degeneration of neurons. Therapeutic strategies were used to inhibit muta...

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Literature Corpus work
0fe4871b-13da-5650-86cb-7102cee6f7e1
DOI
10.21203/rs.3.rs-88222/v1
Open publication

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