In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells
Akshara Sakunthala Velmurugan, Kiriaki Paschoudi, Jack A. Queenan…·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.