Targeted delivery for neurodegenerative disorders using gene therapy vectors : gene next therapeutic goals

Manisha Singh, Surinder P. Singh, Deepshikha Yadav, Mugdha Agarwal, Shriya Agarwal, Vinayak Agarwal, Geeta Swargiary, Sahil Srivastava, Sakshi Tyagi, Ramneek Kaur, Shalini Mani

Research output: Contribution to journalArticlepeer-review

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.
Original languageEnglish
Pages (from-to)23-42
Number of pages20
JournalCurrent Gene Therapy
Volume21
Issue number1
DOIs
Publication statusPublished - 2021

Keywords

  • Alzheimer's disease
  • Parkinson's disease
  • degeneration
  • gene therapy
  • nervous system
  • retroviruses
  • schizophrenia

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