Genome editing for treatment of JAK2 V617F-driven myeloproliferative neoplasms
Roman M. Doll, Yuqi Shen, Zoë C. Wong, Ramy Slama, Maria Greco, Lai Cheng, Eleni Louka, Weijiao Zhang, Yavor K. Bozhilov, Joseph C. Hamley, Adam C. Wilkinson, Bethan Psaila, Adam J. Mead, James O. J. Davies
This study uses CRISPR-Cas genome editing to selectively inactivate JAK2 V617F mutations in patient cells, showing that heterozygous cells are not oncogene addicted while homozygous cells are eradicated, suggesting genome editing as a potential treatment for MPNs.
bioRxiv · Dec 19, 2025 · PreprintCancer Biology