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 ?-globin gene comprised in the genome thereof, at least
one transgene encoding a functional ?-like globin protein, the said transgene being placed
under the control of the endogenous promoter of the said at least one ?-globin gene

Keywords: Biologic, Cell Therapy, Gene Therapy, Genetic Disorders, Hematological Disorders, Others, Product;Therapeutic
Patent Application number: European Procedure (Patents) (EPA) - 12 Avr. 2019 - 19 305 484.8
Inventors:
AMENDOLA Mario,PAVANI Giulia

Reference:

BIO19153-T1

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Aymeric Empereur
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Rare disease: No
Second indication: No

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