Recent advance and prospect of gene therapy for Huntington's disease
10.3760/cma.j.cn115354-20230610-00320
- VernacularTitle:亨廷顿病基因治疗的研究进展及展望
- Author:
Weimeng HUANG
1
;
Hao LIN
;
Zhimei GUO
;
Lin LU
;
Tengteng WU
;
Li GUO
;
Pingyi XU
Author Information
1. 广州医科大学附属第一医院神经科，广州 510120
- Keywords:
Huntington's disease;
Gene therapy;
Gene editing
- From:
Chinese Journal of Neuromedicine
2023;22(11):1160-1163
- CountryChina
- Language:Chinese
-
Abstract:
Huntington's disease (HD) is an autosomal dominant inherited disease with insidious onset and slow progression, mainly characterized by chorea-like symptom, intelligence decline, and psychiatric abnormalities. Cause of the disease is abnormal expansion of CAG trinucleotide repeat sequences in the first exon of the Huntington gene (HTT) on chromosome 4. Despite the clear etiology, currently, no effective therapeutic measures to control the disease progress is noted, and symptomatic treatment is still the main treatment in clinical practice. This article provides a brief overview of the current clinical trials, clinical challenges, and future development of HD gene therapy to provide references for subsequent related research.
