Neurofibromatosis type 2 (NF2) is a rare disorder caused by variants in NF2 gene. It results in growths that appear in nervous tissue across the body. It is critical we prioritize the development of therapeutics for NF2. This proposal aims to understand how RNA-based therapies can be optimized to target Schwann cells, the major cell type affected in NF2 using induced-pluripotent-stem-cell models derived from NF2-patients. We anticipate our work will provide proof-of-concept that NF2-patient-cells can efficiently uptake/process nanoparticles for the delivery of non-pathogenic NF2 mRNA that will be sufficient to reverse proliferative potential of cells and prevent uncontrolled-growth.
RNA therapies for the treatment of Neurofibromatosis type
Project leader:
Jo Anne Stratton
Sector:
Health
Budget:
100 000,00 $
Start date: 01 November 2025 End date: 31 October 2026