Drs. Jonathan Gootenberg and Omar Abudayyeh’s project aims to increase frataxin protein levels in FA patients by improving translation of the patient’s own FXN mRNA. Using a protein-free trans-splicing platform called PRECISE, the team will replace exon 1 of FXN mRNA with an engineered version containing an optimized 5’ UTR to enhance translation. This approach is designed to restore frataxin protein output 5–10 fold without introducing a new gene or permanently altering DNA. By maintaining natural regulation of the gene, it avoids the risks associated with overexpression and conventional gene therapy. If successful, this strategy could provide a safe and effective way to compensate for the transcriptional deficits that cause FA, potentially improving mitochondrial function and reducing cell damage in affected tissues.
General Research Grant | Gene & Stem Cell Therapy
RNA Writing Trans-Splicing for Enhancing Frataxin Translation
Grant Awarded | Jan 2026
Jonathan Gootenberg, PhD, and Omar Abudayyeh, PhD
Beth Israel Deaconess Medical Center and Brigham and Women’s Hospital
Active
The FARA Grant Program is proud to award a General Research Grant to Jonathan Gootenberg, PhD, of Beth Israel Deaconess Medical Center and Omar Abudayyeh, PhD, of Brigham and Women’s Hospital to develop a novel RNA-based strategy to enhance frataxin production in Friedreich’s ataxia.
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