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Article

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Jan 2017

Monteys Alex Mas, Ebanks Shauna A, Keiser Megan S, Davidson Beverly L

Abstract excerpt

Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused by CAG repeat expansion (>36 repeats) within the first exon of the huntingtin gene. Although mutant huntingtin (mHTT) is ubiquitously expressed, the brain shows robust and early degeneration. Current RNA interference-based approaches for lowering mHTT expression have been efficacious in mouse models, but basal mutant protein...

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Identifiers

PMID: 28129107
DOI: 10.1016/j.ymthe.2016.11.010
PMCID: PMC5363210

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