Children's Tumor Foundation

The project aims to address Neurofibromatosis type 2 (NF2) by restoring physiological NF2 gene function using CRISPR-based gene editing technology. We seek to permanently cure NF2 schwannomas and related nerve sheath tumors by targeting Schwann cells in peripheral nerve sheath tumors. Utilizing an adeno-associated virus (AAV)-based vector system with enhanced delivery capabilities, the project aims to develop a safe and effective therapy approach that can be translated into human clinical trials. Through a stepwise research program, we will optimize the delivery system, develop gene editing machinery, and validate the feasibility of NF2 gene editing therapy in relevant biological models, ultimately laying the groundwork for potential future treatments for NF2 patients.

Cooperating partners

Leibniz Institute on Aging – Fritz Lipmann Institute (FLI)

• Helen Morrison
• Lars Björn Riecken

University of Sydney

• Samantha Ginn