Recently, we brought together nine research teams for our first-ever in-person Gene Therapy Initiative Annual Meeting.

The Gilbert Family Foundation is excited to announce a new round of three-year grants in the Gene Therapy Initiative to support the development of curative therapies that address the underlying cause of NF1.

GFF's Gene Therapy Initiative seeks to explore and develop gene-targeting therapeutic strategies for neurofibromatosis type 1 (NF1) that address the underlying cause of the disease. Targeting the mutated gene itself could yield a more effective, comprehensive response for a greater range of patients.

While more than 1000 different mutations of the neurofibromatosis type 1 (NF1) gene have been identified to cause the disease, 1 in 5 are classified as "nonsense mutations". Several projects in GFF's Gene Therapy Initiative are exploring and identifying potential therapeutics to address NF1 nonsense mutations.

The Gilbert Family Foundation's seeks to eradicate neurofibromatosis type 1 (NF1). With our first initiative, we plan to accelerate the development of curative therapies that address the underlying genetic abnormalities in NF1 patients.