Molecular genetics of optic glioma: Lessons learned from neurofibromatosis-1 genetically engineered mice

Neurofibromatosis type 1 is an inherited cancer predisposition syndrome in which 15-20% of children develop optic gliomas. Most of these tumors form along the optic nerve and chiasm, where they can cause decreased visual function and endocrine dysfunction. Advances in mouse modeling of this disease have created tractable models to study the role of specific genetic alterations, deregulated intracellular signaling pathways and non-neoplastic cells in the development of optic gliomas. These small-animal models have been used as platforms to discover new targets for drug design and to evaluate the efficacy of these potential anticancer treatments prior to clinical trials in children with optic glioma.