This supplemental funding builds on the groundwork laid by the 2022 grant awarded to Drs. Markus Grompe and Raymond Monnat, titled Oral Mucosal Gene Therapy as a Prevention for FA-Associated Cancers. The proposed research focuses on reducing the risk of squamous cell carcinoma (SCC) in individuals with Fanconi anemia (FA) by correcting the FA genetic defect in K14-positive basal stem cells, which are the origin of SCC.

People with FA are at much higher risk of developing SCC because their K14 basal stem cells in the squamous epithelium are particularly vulnerable. Gene therapy that corrects this genetic defect directly in these cells offers a potentially transformative approach to cancer prevention. This research seeks to determine if gene-corrected oral stem cells have a selective advantage, allowing healthy cells to outcompete those with mutations. The results will help assess the viability of targeted gene therapies for FA, advancing a new and highly promising strategy to reduce cancer risk in this population.