In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells
Akshara Sakunthala Velmurugan, Kiriaki Paschoudi, Jack A. Queenan, Sucheol Gil, Aidan Maynard, Charlie E Fontana, Reva Sharma, Anika Long, Chang Long Li, Hongjie Wang, Anna Kate Anderson, Aphrodite Georgakopoulou, Nikoletta Psatha, Maria Giannaki, Efthymia Vlachaki, , Karthik V. Karuppusamy
This study demonstrates in vivo gene therapy for hemoglobinopathies using EPOR editing to enhance the expansion of gene-corrected erythroid cells without drug selection.
bioRxiv · Sep 17, 2026 · PreprintBioengineering