BioengineeringbioRxiv
Safety and efficacy study of CRISPR/Cas9 treatment of sickle cell disease in clinically relevant conditions highlights disease-specific response
G. Frati, M. Brusson, G. Sartre…·A. Miccio
This study evaluates the safety and efficacy of CRISPR/Cas9-mediated HbF reactivation in sickle cell disease by testing it in patient-derived HSPCs and shows it is effective but has off-target effects.
DOI 10.1016/j.ymthe.2024.07.015