Development of a peptide that restores the mutant NF1-Ras interaction

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

Current Stage

Discovery

Discovery
In Vivo Proof of Concept
IND Enabling
Clinical Trial Phase 1
Clinical Trial Phase 2
Clinical Trial Phase 3