CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
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United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Scope and Contents
Contents
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 expressi...
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Full title
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5363210
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5363210
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1016/j.ymthe.2016.11.010