NF1-associated optic glioma

Gliomas are the most common central nervous system tumor encountered in individuals with NF1. These tumors are typically low-grade gliomas (pilocytic astrocytomas) involving the optic nerve, chiasm, and tracts (optic pathway gliomas), but they may also develop in the hypothalamus or brainstem of children with NF1. Over the past decade, several Nf1 genetically-engineered mouse models of optic glioma have been established. These instructive preclinical mouse strains have revealed new insights into the role of the NF1 protein (neurofibromin) in astrocyte and neural stem cell growth regulation as well as the importance of the tumor microenvironment and genomic modifiers to gliomagenesis and tumor maintenance. In this chapter, we review these new findings and discuss their implications for future therapeutic drug design.