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CTF funds $1 million in NF2 Gene Therapy over the next three years  

By February 15, 2023December 18th, 2023NF2-SWN, Science & Research

The Children's Tumor Foundation is proud to announce a collaboration between Drs. Helen Morrison, Fritz Lipmann Institute Germany, and Samantha Ginn, University of Sydney. Employing the power of recent scientific advances, in particular CRISPR-based gene editing, we will attempt to restore physiological NF2 gene function within cells to permanently cure the cause for NF2 schwannomas. Furthermore, this project uses an adeno-associated virus (AAV)-based vector system and takes advantage of newly developed capsids with superior delivery to primary human Schwann cells, an important prerequisite for achieving efficient genome editing rates.