GENE THERAPY IN REGARDS TO ATAXIA TELANGIECTASIA MUTATED

ABSTRACT

In this investigation, observations of previously published studies were utilized to review, compare, contrast, and analyze different modern methods of genetic therapy with specific regard to the genetic mutation of Ataxia Telangiectasia. The purpose of this research was to identify whether or not gene therapy would be effective in treating this mutation, and further conclude what method of gene therapy would potentially be the best approach to treatment. 
To achieve this conclusion, multiple aspects of different approaches to genetic therapy were researched and evaluated- including viral vectors, nonviral vectors, and gene editing technology called CRISPR / Cas9. Their structures, processes, limitations, and viability to be applied to ATM treatment were all mainly approached during each evaluation.
What resulted from this thorough observation was a conclusion that in any specific case, multiple pros and cons must be considered to decide which method would be best suited for treating it, and in the case of treating ATM in humans, non-viral techniques would likely be preferred among potential researchers. For future preclinical or clinical studies, depending on the subjects, either viral or nonviral approaches hold great potential to yield positive results.