Children with neurofibromatosis type 1 (NF1) are prone to the development of benign brain tumors that affect the optic pathway and result in vision loss. While current chemotherapy is effective at halting tumor growth, few patients regain vision. For this reason, it is imperative to identify new treatments that prevent or restore vision loss from these tumors.
Research over the past 15 years in the Gutmann laboratory has demonstrated that mice engineered with NF1 gene mutations develop optic pathway gliomas (OPGs) and tumor-associated vision loss. In this project, we plan to use a novel discovery strategy to identify a core molecular signature of vision recovery following treatment in mice, and employ these findings to evaluate promising candidates in preclinical studies in genetically engineered mice with OPGs and vision loss. Leveraging the VRI team science paradigm, discoveries made using these mice will be validated in other experimental paradigms developed in the Reh, Zack, and Young laboratories. In addition, novel findings emanating from other team efforts will be evaluated in these NF1 OPG mice. Taken together, we aim to identify targeted therapeutic strategies that prevent or ameliorate vision loss in children with NF1-associated OPGs.
Investigators
David Gutmann, MD, PhD
Washington University in St. Louis
David Gutmann, MD, PhD
Washington University in St. Louis