The clinical severity of ?-hemoglobinopathies is alleviated by the co-inheritance of genetic mutations causing a sustained fetal ?-globin chain […]

The present invention relates to a genetically modified hematopoietic stem cell(HSC) comprising, in at least one ?-globin gene comprised in the genome […]

The normal development of red blood cells requires a coordinated synthesis of the hemoglobin (Hb) subunits, the ?- and ?-globins in the case of adult […]