Utilizing gene-editing and stem cell therapy to create a novel treatment plan for Tay-Sachs disease
CWSF · 2026 Disease & Illness
Overview
This project uses technology that allows our genetic code to be edited in order to create a new treatment plan for a genetic disorder called Tay-Sachs disease. This disorder has a 100% fatality rate, and the life-span of patients who inherit it is 2-4 years old. The treatment that I have propsed would prevent the disorder from developing symptoms that cause our neurons to break down, ultimately preventing death. Due to the fact that Tay-Sachs disease is a recessive disorder, both of the parents need to have a gene mutation in order for the child to inherit it. This means that the gene mutation can be tested for early enough that this treatment plan could prevent most symptoms from developing, stopping traits of Tay-Sachs disease like blindness, seizures and loss of muscle control from manifesting in patients with Tay-Sachs disease.
Awards (1)
- Selected for CWSF 2026
Competition history
- CWSF 2026
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