• Professor of Neurology and of Cell and Developmental Biology

MD (1989), University of Poona, India
PhD (1995), Northwestern University

Research in the Opal laboratory focuses on the molecular and cellular basis of neurodegeneration with the goal to develop therapies for diseases that currently have no viable treatment options. Since establishing the lab in 2003, we have been studying the pathogenesis of spinocerebellar ataxia type 1 (SCA1), which causes degeneration, most notably of Purkinje cells, and inexorable decline toward death. We discovered that the angiogenic and trophic vascular endothelial growth factor (VEGF) is transcriptionally repressed very early in SCA1 mice, and replenishing its levels is therapeutic. We also tested recombinant VEGF, which was effective in SCA1 mice, and — in a recent collaboration with the laboratory of Samuel Stupp — developed synthetic VEGF-mimetic nanoparticles that work even more effectively and are less costly to produce. This is one promising path to therapy that we are pursuing with CRN.