Development of novel gene therapy strategies for treatment of all SCN2A Disorders

Awardee: Samuel Young

Institution: University of Iowa

Grant Amount: $61,068.00

Funding Period: February 1, 2023 - January 31, 2024

---

Summary:

SCN2A disorders comprise a complex landscape of both missense and protein-truncating variants, resulting in a diversity of phenotypes that include epilepsy and intellectual disability. Currently, there is no cure for SCN2A Disorders, nor are there methods in development that would provide therapeutic intervention for all forms of SCN2A Disorders. Here, our team proposes proof-of-principle studies that could be beneficial for both missense and protein-truncation cases, providing a single method to treat the entire diversity of SCN2A Disorders.