Understanding Spinocerebellar Ataxia Type 1 – A Devastating Progressive, Degenerative Disease

Dr. Opal is studying a genetic disease called Spinocerebellar Ataxia Type 1 that affects the cerebellar region of the brain. This is a relentless and uniformly fatal disease with no current cure. Our hypothesis is that the vascular growth factor VEGF is decreased in SCA1 cerebella and that some aspects of the disease could be reversed by replenishing VEGF. Interest in VEGF from other branches of medicine increases the likelihood that our promising studies could rapidly bring therapies from bench to bedside for SCA1. The BRF grant will be crucial in providing my lab with the funds to obtain preliminary data to compete for NIH R01 level funding.