Federal Project Grant Award Summary

Florida International University received a $1.32M project grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), effective January 26, 2026, with completion targeted for December 31, 2030. The award supports research to develop CRISPR/dCas9 (dead Cas9)-mediated gene-targeted therapeutic interventions for Friedreich's Ataxia (FRDA), a rare autosomal recessive neurological disorder caused by expanded GAA (guanine-adenine-adenine) repeats in the frataxin gene. The research investigates how targeted histone demethylation at the FXN gene can disrupt heterochromatin, activate base excision repair mechanisms, contract pathogenic GAA repeats, and restore FXN gene expression to alleviate FRDA phenotypes in neural and cardiac cell models.

The project integrates multiple research approaches to advance this therapeutic development pipeline. Specific deliverables include determining whether CRISPR/dCas9-mediated histone demethylation can induce DNA repair and GAA repeat contraction, evaluating specific histone demethylases (KDM4D, KDM6A, and KDM6B) for their efficacy in contracting repeats, and testing human vault nanoparticle-based delivery systems for encapsulating therapeutic components to facilitate targeted gene modification. This multi-disciplinary effort combines molecular biology, genome engineering, and nanomedicine approaches to generate foundational evidence supporting potential future clinical development of a precision medicine therapeutic for this currently untreatable genetic neurological disease.