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CRISPR-Based Genome-Editing Tools for Huntington’s Disease Research and Therapy

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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.

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Funding

This review was supported by the National Key R&D Program of China (2021YFA0805200), the National Natural Science Foundation of China (31970954, 81901289 and 31872779) and the Guangdong Key Laboratory of Non-human Primate Research (2020B121201006).

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  1. Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China

    Yiyang Qin, Shihua Li, Xiao-Jiang Li & Su Yang

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  1. Yiyang Qin
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Correspondence to Su Yang.

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Qin, Y., Li, S., Li, XJ. et al. CRISPR-Based Genome-Editing Tools for Huntington’s Disease Research and Therapy. Neurosci. Bull. 38, 1397–1408 (2022). https://doi.org/10.1007/s12264-022-00880-3

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