Dhruva Singu

How can gene therapy be utilized to target specific mutations in Usher Syndrome, and what are the current successes, limitations, and future directions in developing an effective treatment?

Started Feb. 10, 2025

Abstract or project description

This project is a research paper answering the following question: How can gene therapy be utilized to target specific mutations in Usher Syndrome, and what are the current successes, limitations, and future directions in developing an effective treatment? Usher Syndrome is becoming more and more prevalent globally today and this genetic condition can cause sensorineural hearing loss, vision loss, and in some cases balance issues. Gene Therapy is a promising solution to correcting the mutations associated with Usher Syndrome, and by delivering function copies of various genes associated with the different types of Usher Syndrome, the disorder has the potential to be solved with gene therapy. To complete this project, various existing literature on Usher Syndrome will be analyzed so a solution that effectively works can be reached.