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Nature Medicine|January 30, 2019
Identification of preexisting adaptive immunity to Cas9 proteins in humansCarsten T Charlesworth, Priyanka S Deshpande, Daniel P Dever, et al.Nature Medicine|March 19, 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cellsM Kyle Cromer, Joab Camarena, Renata M Martin, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 1, 2025
A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expressionKirby A Wallace, Trevor L Gerstenberg, Craig L Ennis, et al.Nature Communications|March 24, 2024
Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed RepairJuan A Perez-Bermejo, Oghene Efagene, William M Matern, et al.Pageof 3