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

Targeted delivery for neurodegenerative disorders using gene therapy vectors: Gen next therapeutic goals. Curr Gene Ther. 2020 Aug 17;: Authors: Singh M, Singh SP, Yadav D, Agarwal M, Agarwal S, Agarwal V, Swargiary G, Srivastava S, Tyagi S, Kaur R, Mani S Abstract The technique of gene therapy, ever since its advent nearly fifty years ago, has been utilized by scientists as a po-tential 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 therole that gene therapy can play in combating them. The pathogenesis and the molecular mechanisms specifying the altered gene ex-pression of each of these NDDs has 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 pro-gressive research towards its implementation can provide us with powerful treatment options for disease conditions that have so far been considered as incurable. PMID: 32811395 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research