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LOVOTIBEGLOGÉN AUTOTEMCEL
LOVOTIBÉGLOGENE AUTOTEMCEL
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS (HSCS) OBTAINED FROM MOBILISED PERIPHERAL BLOOD, FROM PATIENTS WITH SICKLE CELL DISEASE. THE CELLS ARE TRANSDUCED WITH BETIBEGLOGENE DAROLENTIVEC, A SELF-INACTIVATING HUMAN IMMUNODEFICIENCY VIRUS (HIV-1)-DERIVED LENTIVIRAL VECTOR ENCODING A T87Q-MUTATED FORM OF THE HUMAN HEMOGLOBIN SUBUNIT BETA (HBB, BETA-GLOBIN) GENE UNDER THE CONTROL OF A HUMAN BETA-GLOBIN PROMOTER AND A 3' BETA-GLOBIN ENHANCER; THE VECTOR GENOME IS FLANKED BY 5' AND 3' LONG TERMINAL REPEATS (LTRS) AND CONTAINS A PSI PACKAGING SIGNAL, A REV RESPONSE ELEMENT (RRE), AND TWO POLYPURINE TRACT (PPT) SEQUENCES. THE CD34+ CELL POPULATION WAS ENRICHED USING IMMUNOMAGNETIC MICROBEADS, AND THEN CULTURED IN A STEM CELL GROWTH MEDIUM, SUPPLEMENTED WITH RECOMBINANT HUMAN CYTOKINES SUCH AS STEM CELL FACTOR (SCF), FMS-RELATED TYROSINE KINASE 3 LIGAND (FLT-3L) AND THROMBOPOIETIN (TPO). THE CELLS CONSIST OF CD34+ CELLS (MORE OR EQUAL TO 70%), OF WHICH MORE OR EQUAL TO56% EXPRESS THE TRANSGENE