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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

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5363210

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

About this item

Full title

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

Publisher

United States: Elsevier Inc

Journal title

Molecular therapy, 2017-01, Vol.25 (1), p.12-23

Language

English

Formats

Publication information

Publisher

United States: Elsevier Inc

More information

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...

Alternative Titles

Full title

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

Identifiers

Primary Identifiers

Record Identifier

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

Other Identifiers

ISSN

1525-0016

E-ISSN

1525-0024

DOI

10.1016/j.ymthe.2016.11.010

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