Targeted Delivery for Neurodegenerative Disorders Using Gene Therapy Vectors: Gene Next Therapeutic Goals

Author(s): Manisha Singh, Surinder P. Singh, Deepshikha Yadav, Mugdha Agarwal, Shriya Agarwal, Vinayak Agarwal, Geeta Swargiary, Sahil Srivastava, Sakshi Tyagi, Ramneek Kaur, Shalini Mani*

Journal Name: Current Gene Therapy

Volume 21 , Issue 1 , 2021

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Graphical Abstract:


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.

Keywords: Gene therapy, neurodegenerative diseases, viral vectors, nonviral vectors, Alzheimer disease, Parkinson disease, schizophrenia retroviruses.

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Article Details

Year: 2021
Published on: 17 August, 2020
Page: [23 - 42]
Pages: 20
DOI: 10.2174/1566523220999200817164907
Price: $65

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