top of page
gt 1.jpeg

GENE THERAPY IN REGARDS TO ATAXIA TELANGIECTASIA MUTATED

A Literary Review

Home: Welcome

ABOUT ME

IMG_1258_edited.jpg

RACHEL KLUDY

High School Class of 2024

Hi! I'm Rachel, a high school freshman at American Heritage School in Plantation, Florida. I'm in a premedical program as well as a scientific research class.

THE PROJECT

Gene Therapy in Regards to Ataxia Telangiectasia Mutated (ATM): A Review

This literary research project was completed in 2020. It means a lot to me personally, as ATM is a subject that affects me and my family as well as countless others, but goes unfortunately unnoticed in many circumstances.

capsid_rainbow_ListingImage-4096x2731.jp
Home: Work

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.

Home: About Me
Home: Video

CONTACT

954-330-0053

Home: Contact

IMAGE CREDITS

Header Image:

Pasieka, Alfred. Gene Therapy, Nature.com, www.nature.com/gt/.

"The Project" Image:

“Synthetic AAV Capsids for Advanced Gene Therapy.” Wyss Institute, Harvard University, wyss.harvard.edu/technology/synthetic-aav-virus-capsids-for-advanced-gene-therapy/.

Home: Text
bottom of page