Genetically-Engineered Mouse Models of Cancer
A critical first step towards identifying new treatments for people affected with brain tumors, nerve sheath tumors (neurofibromas and sarcomas) or cognitive delays is the development of robust informative preclinical mouse models of human disease.
Over the past 20 years, our laboratory has generated numerous genetically-engineered mouse models of NF1-associated brain tumors, NF1-associated attention and learning deficits and sporadic pediatric low-grade brain tumors. We have extensively credentialed these models with respect to their ability to accurately represent the human counterparts, and employed them to discover and validate new promising treatments for NF1-associated learning deficits and optic glioma. Several of these drugs are now in clinical trials for children with NF1. In addition, these models provide highly instructive platforms to critically dissect the pathogenesis of human disease.
Current projects in the Gutmann research laboratory are focused on developing additional mouse models of Nf1 optic gliomas, brainstem gliomas and neurofibromas, as well as sporadic non-NF1-associated low-grade pediatric brain tumors.