Neurofibromatosis type 1 (NF1) is a cancer predisposition syndrome, and somaticNF1 mutations are also common in tumours. The neurofibromin protein (encoded by NF1) is a RAS GTPase-activating protein that activates RAS–MAPK signalling when lost. This Review discusses NF1 disease, neurofibromin signalling pathways and recent developments in NF1 therapeutics.
- Nancy Ratner
- Shyra J. Miller