Gene Therapy to Treat Neurofibromatosis using a Foamy Viral Vector

Neurofibromatosis type 1 (NF1) is a common genetic condition marked by features such as café-au-lait skin spots, freckling in the underarm and groin areas, benign eye tumors, bone abnormalities, and an increased risk of developing tumors along nerves. Current treatments mainly include surgery and the drug selumetinib for plexiform neurofibromas in children. Unfortunately, many tumors cannot be fully removed because of their location, and even when surgery is possible, tumors often grow back. Gene therapy using Foamy Virus (FV) vectors offers a promising new approach. These vectors can deliver a healthy copy of the NF1 gene into affected cells, restore production of the neurofibromin protein, and potentially stop tumor growth. This proposal aims to advance FV-based gene therapy for NF1-related tumors and move it closer to clinical use.