VEGF as therapy for SCA1

We are 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. We are currently focusing on using mouse models in SCA1. However, we hope that our studies will provide the impetus to initiate clinical trials for patients with SCA1 and perhaps other ataxias.