脊髓性肌萎缩
遗传增强
疾病
医学
基因传递
肌萎缩侧索硬化
生物信息学
神经科学
进行性肌萎缩
亨廷顿病
生物
基因
基因组编辑
癌症研究
转基因
载体(分子生物学)
病理
遗传学
作者
Manisha Singh,Surinder P. Singh,Deepshikha Yadav,Mugdha Agarwal,Saurabh Agarwal,Vinayak Agarwal,Geeta Swargiary,Sahil Srivastava,Sonia Tyagi,Rajvir Kaur,Shalini Mani
标识
DOI:10.2174/1566523220999200817164907
摘要
The technique of gene therapy, ever since its advent nearly fifty years ago, has been utilized by scientists as a potential treatment option for various disorders. This review discusses some of the major neurodegenerative diseases (NDDs) like Alzheimer's disease (AD), Parkinson's Disease (PD), Motor neuron diseases (MND), Spinal Muscular Atrophy (SMA), Huntington's Disease (HD), Multiple Sclerosis (MS), etc. and their underlying genetic mechanisms along with the role that gene therapy can play in combating them. The pathogenesis and the molecular mechanisms specifying the altered gene expression of each of these NDDs have also been discussed in elaboration. The use of gene therapy vectors can prove to be an effective tool in the field of curative modern medicine for the generations to come. Therefore, consistent efforts and progressive research towards its implementation can provide us with powerful treatment options for disease conditions that have so far been considered as incurable.
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