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.

Other Grants

Lindsay M. De Biase, Ph.D., University of California Los Angeles
The role of microglial lysosomes in selective neuronal vulnerability
Synapses, the sites of signaling between neurons in the brain, play essential roles in learning, memory, and the health of neurons themselves. An enduring mystery is why some neurons are…
How the nervous system constructs internal models of the external world
As animals navigate their environments, they construct internal models of the external sensory world and use these models to guide their behavior. This ability to incorporate ongoing sensory stimuli into…
Xiaojing Gao, Ph.D., Stanford University
When Neural Circuits Meet Molecular Circuits: Quantitative Genetic Manipulation with Single-cell Consistency
Cells are the building blocks of our bodies. We get sick when the cells “misbehave”. The way modern gene therapies work is to introduce genes, fragments of DNA molecules that…
Rafiq Huda, Ph.D., Rutgers University
Conducting the orchestra of movement—functional role of striatal astrocytes in health and disease
Movement requires coordinated activity across a large brain-wide network. The striatum is a particularly important part of this circuit; it integrates motor-related information from many distinct brain regions to regulate…