PHARMASPIRE - Volume 10, Issue 3, July - September, 2018
Pages: 107-112
Date of Publication: 14-Jun-2022
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Gene therapy as a potential tool for neurodegenerative disorders: Possible highlights
Author: Raman Kumar Tripathi, Arti Rana, Shamsher Singh
Category: Pharmaceutics
Abstract:
Gene therapy is the transmission of genes into the patient to treat the illness. Gene can be transferred through somatic and germline techniques. In the somatic type of gene therapy, the genes are transmitted in the germ cells or stem cells whereas in germline gene therapy the genes are transmitted in the DNA or genome so it can pass to the offspring. The carriers used in this purpose are viral and non-viral vectors. Basically, these viral and non-viral carriers help to transfer the genes which further show its expression either by synthesizing proteins or causing mutation of DNA. Gene therapy is used in the cure or management of monogenic disorders and polygenic disorders; moreover, it is also a potential target for neurological disorders like in Alzheimer disease (AD) using gene therapy it is possible to inhibit, the progression of amyloidbeta peptide, a protein which causes amyloid plaques. Similarly, in Parkinson disease, the transfer of Neurturin, an analog of glial cell line-derived neurotrophic factor helps in survival of the dopaminergic neurons. In Huntington’s disease (HD), ASOS is used in the gene silencing which forms the protein known as Huntingtin. The HD may also be cured by the Casper cas 9 therapy. It would edit the DNA so that it will not make Huntingtin protein by transcription, thus treating the disease. By using this technique it’s useful to treat various disorders as genetic predisposition or in which the available drugs are unable to prevent or treat such problems as neurological and some of the non-neurological.
Keywords: Alzheimer disease, Crispr Cas9 Therapy, gene therapy, Parkinson’s disease
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