PMID- 35608753
OWN - NLM
STAT- MEDLINE
DCOM- 20221121
LR  - 20231102
IS  - 1995-8218 (Electronic)
IS  - 1673-7067 (Print)
IS  - 1995-8218 (Linking)
VI  - 38
IP  - 11
DP  - 2022 Nov
TI  - CRISPR-Based Genome-Editing Tools for Huntington's Disease Research and Therapy.
PG  - 1397-1408
LID - 10.1007/s12264-022-00880-3 [doi]
AB  - 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.
CI  - (c) 2022. Center for Excellence in Brain Science and Intelligence Technology, 
      Chinese Academy of Sciences.
FAU - Qin, Yiyang
AU  - Qin Y
AD  - Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau 
      Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.
FAU - Li, Shihua
AU  - Li S
AD  - Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau 
      Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.
FAU - Li, Xiao-Jiang
AU  - Li XJ
AD  - Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau 
      Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.
FAU - Yang, Su
AU  - Yang S
AD  - Guangdong Key Laboratory of Non-human Primate Research, Guangdong-Hongkong-Macau 
      Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China. 
      syang33@jnu.edu.cn.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20220524
PL  - Singapore
TA  - Neurosci Bull
JT  - Neuroscience bulletin
JID - 101256850
SB  - IM
MH  - Humans
MH  - Gene Editing
MH  - *Huntington Disease/genetics/therapy
MH  - CRISPR-Cas Systems/genetics
MH  - *Neurodegenerative Diseases
PMC - PMC9672252
OTO - NOTNLM
OT  - Animal models
OT  - CRISPR
OT  - Huntington's disease
COIS- The authors declare no competing financial interests.
EDAT- 2022/05/25 06:00
MHDA- 2022/11/22 06:00
PMCR- 2023/11/01
CRDT- 2022/05/24 11:19
PHST- 2021/12/02 00:00 [received]
PHST- 2022/03/17 00:00 [accepted]
PHST- 2022/05/25 06:00 [pubmed]
PHST- 2022/11/22 06:00 [medline]
PHST- 2022/05/24 11:19 [entrez]
PHST- 2023/11/01 00:00 [pmc-release]
AID - 10.1007/s12264-022-00880-3 [pii]
AID - 880 [pii]
AID - 10.1007/s12264-022-00880-3 [doi]
PST - ppublish
SO  - Neurosci Bull. 2022 Nov;38(11):1397-1408. doi: 10.1007/s12264-022-00880-3. Epub 
      2022 May 24.