In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells
In vitro studies in an erythroid progenitor cell line and primary CD34+ cells demonstrated that the EPORW439* variant conferred a strong proliferative advantage to therapeutically modified erythroid progenitors, establishing a strategy to amplify the therapeutic benefit of otherwise limited in vivo HSPC gene editing fo...
Akshara Sakunthala Velmurugan, K. Paschoudi, Jack A. Queenan et al.
· bioRxiv · 0 citations