Correction of beta-thalassemia phenotype by genetically engineered hematopoietic stem cell

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

Keywords: Biologic, Cell Therapy, Gene Therapy, Genetic Disorders, Hematological Disorders, Others, Product;Therapeutic



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Aymeric Empereur
Aymeric Empereur
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