The Detroit-based Gilbert Family Foundation has announced a three-year, $12 million initiative aimed at accelerating the development of curative therapies that address the underlying genetic abnormalities in neurofibromatosis type 1 (NF1) patients.
NF1 causes tumors to form on nerves in the body, which can lead to disfigurement, blindness, and cancer and/or result in learning disabilities, bone deformities, and cardiovascular issues. While NF1 drug development has focused on addressing the symptoms, the foundation's initiative is aimed at developing therapies that address the underlying genetic causes of NF1 — including gene replacement, gene editing, RNA editing, exon skipping, and nonsense mutation suppression.
Based on the belief that directly repairing, replacing, or bypassing the NF1 gene mutation is an important piece of any NF cure, the foundation awarded grants to eight research teams working to identify promising gene therapy techniques. The lead researchers and their institutions are Charles Gersbach (Duke University); Eric Pasmant (Paris Descartes University); Andre Leier, David Bedwell, Deanne Wallis, and Robert Kesterson (University of Alabama); and Allan Jacobson and Miguel Sena-Esteves (University of Massachusetts).
"We have been deeply impacted ever since our oldest son Nick was diagnosed with NF1 as a toddler,” said Jennifer Gilbert, “and believe this research will benefit numerous families affected by NF1 around the globe, as well as our son.”
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