CORRECTION OF ΒETA-THALASSEMIA PHENOTYPE BY GENETICALLY ENGINEERED HEMATOPOIETIC STEM CELL

摘要

The present invention relates to a genetically modified hematopoietic stem cell (HSC) comprising, in at least one α-globin gene comprised in the genome thereof, at least one transgene encoding a functional β-like globin protein, the said transgene being placed under the control of the endogenous promoter of the said at least one α-globin gene.