BioengineeringMolecular TherapyPeer-reviewed
Safety and efficacy study of CRISPR/Cas9 treatment of sickle cell disease in clinically relevant conditions highlights disease-specific response
Giacomo Frati, Mégane Brusson, Gilles Sartre…·Annarita 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.
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