Swarna Rishika Suresh

Evaluating the efficacy of genetic modifications as potential therapeutics for neurodegenerative diseases

Started Apr. 24, 2024

Abstract or project description

Neurodegenerative diseases, including Alzheimer’s disease, Parkinson's disease, amyotrophic lateral sclerosis, and spinal muscular atrophy are prevalent neurological conditions affecting millions worldwide. These disorders lead to the degeneration of neurons and muscles, which can result in a slow death without treatment. Recently, gene therapy, a technique to modify DNA, RNA, and protein output in these diseases has emerged as a potential solution. This review evaluates the efficacy of gene therapies in treating neurodegeneration and addresses the lack of awareness of gene therapeutic success in both the scientific community and the general public. In this review, the PubMed and Google Scholar databases were used to find and analyze 36 peer-reviewed articles and one thesis. The manuscripts were analyzed to ensure each study was either a randomized or clinical trial. There is a wide variety of gene therapies existing for each disorder, with varying degrees of progress. Whereas Alzheimer’s and Parkinson’s have few clinical trials, SMA and ALS have relatively more (due to the nature of these diseases). Though many of these therapies successfully targeted pertinent biomarkers, they were accompanied by adverse immune effects. Furthermore, the usage of an AAV vector was often safer than the simple delivery of a gene or editing components. Ultimately, science has made major strides in gene therapeutic solutions for neurodegeneration, with some even available to people. However, more work needs to be done in order to first test these components on humans and then deliver them to the general public while maintaining minimal adverse effects.