Gabriel Rui
Class of 2025
About
Projects
- "How can CRISPR-cas be applied to treating genetic diseases caused by repeat expansions such as Huntington’s Disease, Friedrich Ataxia, and Fragile X Syndrome?" with mentor Miwa (Sept. 6, 2024)
Project Portfolio
How can CRISPR-cas be applied to treating genetic diseases caused by repeat expansions such as Huntington’s Disease, Friedrich Ataxia, and Fragile X Syndrome?
Started May 13, 2024
Abstract or project description
About 1 in 3,000 people are affected by a genetic disease caused by repeat expansions in their DNA. Historically, there have been no treatments for these diseases other than symptom management, however, with the advancement of gene editing, most recently CRISPR-cas, new tools and methods are now available to address these diseases. Here, I will discuss different CRISPR-cas methods being studied and used for treating repeat expansion diseases, some of the most prevalent being Huntington’s Disease, Friedrich Ataxia, and Fragile X Syndrome. I will review the latest theories and clinical trials on such treatments, then address some of the complications that are holding people back from successfully treating these diseases with CRISPR-cas, and conclude with the future direction of this field.