CRISPR-Based Genome-Editing Tools for Huntington's Disease Research and Therapy.
10.1007/s12264-022-00880-3
- Author:
Yiyang QIN
1
;
Shihua LI
1
;
Xiao-Jiang LI
1
;
Su YANG
2
Author Information
1. Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.
2. Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China. syang33@jnu.edu.cn.
- Publication Type:Review
- Keywords:
Animal models;
CRISPR;
Huntington’s disease
- MeSH:
Humans;
Gene Editing;
Huntington Disease/therapy*;
CRISPR-Cas Systems/genetics*;
Neurodegenerative Diseases
- From:
Neuroscience Bulletin
2022;38(11):1397-1408
- CountryChina
- Language:English
-
Abstract:
Huntington's disease (HD) is an autosomal dominantly-inherited neurodegenerative disease, which is caused by CAG trinucleotide expansion in exon 1 of the Huntingtin (HTT) gene. Although HD is a rare disease, its monogenic nature makes it an ideal model in which to understand pathogenic mechanisms and to develop therapeutic strategies for neurodegenerative diseases. Clustered regularly-interspaced short palindromic repeats (CRISPR) is the latest technology for genome editing. Being simple to use and highly efficient, CRISPR-based genome-editing tools are rapidly gaining popularity in biomedical research and opening up new avenues for disease treatment. Here, we review the development of CRISPR-based genome-editing tools and their applications in HD research to offer a translational perspective on advancing the genome-editing technology to HD treatment.
