FA – Sanjay Bidichandani, M.B.B.S., Ph.D.

FA - Sanjay Bidichandani, M.B.B.S., Ph.D.
“We believe that this project will enhance our understanding of the molecular pathogenesis of Friedreich’s ataxia, and will have important implications for the development of a specific therapy,” Sanjay Bidichandani says.

CMRI Claire Gordon Duncan Chair in Genetics and Professor of Pediatrics Sanjay Bidichandani, at University of Oklahoma Health Sciences Center in Oklahoma City, was awarded an MDA research grant totaling $300,000 over three years to address clinically and scientifically important questions regarding the use of existing and novel HDAC inhibitors to increase levels of the frataxin protein in Friedreich’s ataxia (FA). Bidichandani and colleagues recently found that the mechanism of gene silencing in FA involves shutting off the FXN gene promoter, a region of the gene that typically controls when and how much a gene should be turned on. They are now working to identify therapeutic molecules that will effectively reverse FXN promoter silencing in FA, and to determine the precise mechanism by which promoter inactivation and reactivation occurs.

Funding for this MDA research grant began Aug. 1, 2015.