摘要
神经系统遗传病种类繁多，至今大多不可治愈。随着分子生物学技术的不断发展，基因增补、外显子跳跃、增强剪接、动态突变修正、毒性表达产物清除等基因治疗策略在神经遗传病的治疗中具有良好的应用前景。
Most of the neurogenetic disorders could not be cured until now. With the development of molecular biological techniques, gene therapies show brilliant application prospects in neurogenetic disorders, which contain gene supplementation, exon skipping, splicing enhancement, dynamic mutation correction and toxic expression product elimination, and so on.
作者
王柠
赵淼
陈万金
Wang Ning;Zhao Miao;Chen Wanjin(Department of Neurology,the First Affiliated Hospital of Fujian Medical University,Fuzhou 350001,China)
出处
《中华神经科杂志》
CAS
CSCD
北大核心
2018年第11期857-862,共6页
Chinese Journal of Neurology
基金
国家自然科学基金资助项目（U1505222）
关键词
神经遗传病
基因治疗
策略
前景
Neurogenetic diseases
Gene therapy
Strategy
Prospect
