What is NF?
Neurofibromatosis, also known as NF, is a genetic disease that causes tumors to grow on nerve pathways anywhere in the body. There are three types of neurofibromatoses – NF1, NF2, and Schwannomatosis. NF1 is the most common type, affecting approximately 1 in 2,500 births, and is the primary focus of our research efforts.
NF1 is characterized by multiple café-au-lait (light brown) skin spots, freckling in the armpits or groin, and/or neurofibromas (small benign growths) on or under the skin. About 50% of people with NF1 also have learning challenges. Tumors may develop in the brain, on the spinal cord, and/or on nerves all throughout the body. While NF1 tumors are generally not cancerous, they may cause significant deformities and health issues such as blindness. Additionally, benign NF1 tumors sometimes become malignant.
The key to finding improved treatments and cures is to establish models of NF1 and implement drug discovery systems that are closer to the true NF1 condition in a human patient. NGNRI aims to pave the way for more personalized health care by leveraging next generation technologies, including ‘organoids’ or organ-like assemblies grown in a lab environment, and advancing leading research.
The Approach
NGNRI combines a state-of-the-art technology platform with a unique guest researcher model. This allows researchers from around the world to launch fully supported parallel labs at NGNRI, gaining access to expert teams and advanced tools to accelerate their work without leaving their home institutions.