Award
NIH Reporter #3R01NS135254-03S1
Alternative Splicing and Development of Small Molecule Therapeutics in CAG Expansion Spinocerebellar Ataxias
Recipient
State University of New York at Albany
Award Amount
$63,755.00
Ceiling
$63,755.00
Awarded
September 03, 2026
Identifier
3R01NS135254-03S1
This project aims to explore the role of alternative splicing in CAG expansion SCAs and develop small molecule therapeutics targeting these mechanisms. The research involves screening FDA-approved drugs and natural products, analyzing transcriptomic signatures, and validating biomarkers in cell and animal models to facilitate clinical translation.
Description
The project focuses on investigating the role of alternative splicing in spinocerebellar ataxias (SCAs), a group of rare neurological disorders characterized by lack of balance and coordination. The research aims to identify therapeutic compounds, including FDA-approved small molecules and natural products, that can modulate CAG repeat expansions and associated splicing dysregulation. The study leverages cell models and transcriptomic analyses to discover biomarkers and therapeutic targets, with the goal of accelerating clinical testing of novel treatments.