Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9-Reference-Cited by-同舟云学术

Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9

Published:2022-10-10 Issue:19 Volume:7 Page:
ISSN:2379-3708
Container-title:JCI Insight
language:en
Short-container-title:

Author:

Shin Jun Wan,Hong Eun Pyo^ORCID,Park Seri S.,Choi Doo Eun,Seong Ihn Sik,Whittaker Madelynn N.^ORCID,Kleinstiver Benjamin P.^ORCID,Chen Richard Z.^ORCID,Lee Jong-Min

Funder

NINDS

National Institute of Health

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://insight.jci.org/articles/view/141042/files/pdf

Reference93 articles.

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2. Huntington disease

3. CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion

4. Identification of Genetic Factors that Modify Clinical Onset of Huntington’s Disease

5. Genetic Modifiers of Huntington’s Disease (GeM-HD) Consortium, et al. Huntington’s disease onset is determined by length of uninterrupted CAG, not encoded polyglutamine, and is modified by DNA maintenance mechanisms [preprint]. https://doi.org/10.1101/529768 Posted on bioRxiv January 24, 2019

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