Federal Project Grant Award Summary

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a Project Grant totaling $619,977 to The Regents of the University of Colorado (University of Colorado-Denver) on May 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). This research initiative will investigate patterns and mechanisms of repeat instability in Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS), a late-onset neurological condition caused by trinucleotide repeat expansions at the FMR1 locus. The project will employ cutting-edge sequencing and analytic approaches to interrogate neurogenetic mechanisms of FMR1 regulation directly in human tissue, addressing a critical gap in understanding the clinical heterogeneity of FXTAS and other fragile X-related conditions. Research activities will be conducted in Aurora, Colorado, with a project completion target of January 31, 2031.

The anticipated deliverables include elucidation of genetic and cellular mechanisms mediating FMR1 repeat instability, with broader applicability to neurologic repeat expansion disorders. These research findings are expected to accelerate therapeutic development and improve clinical care for FXTAS patients, as no therapeutic interventions currently exist for this condition or related fragile X disorders. Given the relatively high prevalence of the FMR1 premutation in the general population, this research addresses an unmet clinical need by leveraging human-based research models to overcome limitations of historical approaches utilizing animal model systems.