Emerging therapeutic approaches of neurogenetic diseases
10.3760/cma.j.cn113694-20220418-00307
- VernacularTitle:神经遗传病治疗的新希望
- Author:
Ruojie HE
1
;
Xiaoli YAO
Author Information
1. 中山大学附属第一医院神经科 广东省重大神经疾病诊治研究重点实验室 国家临床重点专科和国家重点学科，广州510080
- Keywords:
Genetics, medical;
Nervous system diseases;
Clinical trial, phase Ⅰ;
Clinical trial, phase Ⅱ;
Clinical trial, phase Ⅲ;
Gene therapy
- From:
Chinese Journal of Neurology
2022;55(7):671-681
- CountryChina
- Language:Chinese
-
Abstract:
Neurogenetic diseases are a group of hereditary diseases that predominantly affect the nervous system, including the central nervous system, peripheral nerves, and muscles. The clinical manifestations are complex, and the treatments are rather difficult. In recent years, with the in-depth study of the etiology and pathogenesis of neurogenetic diseases, gene therapeutic approaches developed for mutant genes, such as antisense oligonucleotides, RNA interference, adeno-associated virus-mediated gene transduction, small molecule compounds with gene modification effects, and clustered regularly interspaced short palindromic repeats/Cas9 gene editing strategies are gradually transformed into clinical applications. The drugs with potential therapeutic effects are developed in according to the promising targets within the pathogenic mechanism of diseases. These emerging therapeutic approaches provide innovative prospects for patients with neurogenetic diseases.
