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, B. MLAYAH, T. FELIX, A. Chalumeau, P. Antoniou, G. Hardouin, J.-P. Concordet, O. Romano, G. Turchiano, 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.
bioRxiv · Jan 14, 2024Bioengineering