Federal Grant Award Summary

The University of Florida's Division of Sponsored Research received a $423,750 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, effective March 16, 2026, through February 28, 2031. The award supports research to identify approved drugs for treating Brain-Derived Neurotrophic Factor (BDNF) insufficiency, a condition affecting individuals with WAGR (Wilms Tumor, Aniridia, Genitourinary Abnormalities, and Mental Retardation) syndrome. The project focuses on developing therapeutic interventions to increase BDNF expression in patients exhibiting BDNF haploinsufficiency-related complications, including severe obesity, intellectual disability, autism, and impaired pain perception.

The research team has developed an innovative phenotypic assay utilizing a novel BDNFNLUC mouse strain, in which nano luciferase is inserted into the BDNF locus, enabling highly sensitive and reproducible detection of BDNF expression in 384-well plate formats. This assay platform will be leveraged to screen and identify existing approved drugs capable of enhancing BDNF expression, potentially enabling rapid translational advancement toward clinical applications for WAGR patients with BDNF insufficiency. The work integrates basic neuroscience research with translational drug discovery to address a critical unmet medical need in a rare disease population.