This project focuses on the development of a new strategy for the treatment of NF1. The pathology of NF1 disease primarily originates from the broken interaction between the protein neurofibromin and its binding partner Ras. In our work, we will create a vast library of peptides and use a powerful strategy to uncover those peptides that are able to replace the activity of neurofibromin, leading to deactivation of Ras. We will test the activity of the peptides in cells containing NF1 mutants that have been derived from patients, as well as mouse models of NF1. Since this treatment focuses on the root cause of the NF1, rather than downstream pathways, it promises to treat NF1 effectively and with minimal side effects.
Investigators
Matthew Hartman, PhD
Virginia Commonwealth University
Said Sebti, PhD
Virginia Commonwealth University
Zhihong Wang, MD, PhD
Virginia Commonwealth University
Matthew Hartman, PhD
Virginia Commonwealth University
Said Sebti, PhD
Virginia Commonwealth University
Zhihong Wang, MD, PhD
Virginia Commonwealth University