Using neurofibromatosis-1 to better understand and treat pediatric low-grade glioma.

Relatively little is known about the seminal genetic events that trigger the development of low-grade gliomas in children. Genetically engineered mouse models of the neurofibromatosis-1-inherited tumor predisposition syndrome have identified key intracellular growth control pathways, defined the contribution of the tumor microenvironment to glioma growth, and helped researchers understand the genetic basis for glioma susceptibility. In addition, genetically engineered mouse low-grade glioma models have recently been used in preclinical therapeutic studies to evaluate the efficacy of particular biologically based therapies and to define outcome measures.