Research progress in gene therapy for amyotrophic lateral sclerosis
10.3760/cma.j.cn113694-20210826-00590
- VernacularTitle:肌萎缩侧索硬化基因治疗研究进展
- Author:
Zhi MA
1
;
Huihui ZHAO
;
Qi NIU
Author Information
1. 南京医科大学第一附属医院老年神经内科，南京210029
- Keywords:
Amyotrophic lateral sclerosis;
Oligonucleotides, antisense;
Gene therapy;
Adeno-associated virus
- From:
Chinese Journal of Neurology
2022;55(3):260-265
- CountryChina
- Language:Chinese
-
Abstract:
Amyotrophic lateral sclerosis is a neurodegenerative disease caused by the loss of motor neurons in the brain and spinal cord. There is currently no effective cure. The emergence of gene therapy brings hope to treatment, which can be achieved by delivering transgenes to replace or correct defective genes, as well as the expression of neurotrophic factors. The vectors of gene therapy can be viral vectors and non-viral vectors. Lentiviral vectors can be used to deliver therapeutic sequences to motor neurons in the central nervous system. Adeno-associated viruses can effectively mediate gene expression and delivery of neurotrophic factors. Gene editing and antisense oligonucleotides therapy are also perspective treatment options. This article summarizes gene therapy for amyotrophic lateral sclerosis from basic experiments and clinical trials.
