Periodic Reporting for period 1 - EDITSCD (Assessing efficacy and safety of genome EDITing approaches for Sickle Cell Disease)

Sickle cell disease (SCD) is one of the most prevalent monogenic diseases in Europe. A single amino acid substitution in the beta-globin chain of the adult hemoglobin (Hb) drives red blood cell sickling and multi-organ damage. The clinical severity of SCD is alleviated by the...