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MOZAFANCOGÉN AUTOTEMCEL
AUTOLOGOUS CD34+ CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD, TRANSDUCED WITH A SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN FANCONI ANEMIA COMPLEMENTATION GROUP A (FANCA) UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK) PROMOTER; THE CONSTRUCT IS FLANKED BY 5' AND 3' LONG TERMINAL REPEATS (LTRS) AND ALSO CONTAINS A PSI PACKAGING SIGNAL, A REV RESPONSE ELEMENT (RRE), A CENTRAL POLYPURINE TRACT (CPPT) SEQUENCE AND THE WOODCHUCK HEPATITIS VIRUS POSTTRANSCRIPTIONAL REGULATORY ELEMENT (WPRE). THE VECTOR IS PSEUDOTYPED WITH THE ENVELOPE GLYCOPROTEIN (G) OF VESICULAR STOMATITIS VIRUS (VSV). THE CD34+ CELL POPULATION WAS ENRICHED USING IMMUNOMAGNETIC MICROBEADS, AND THE CELLS CULTURED IN BAGS COATED WITH A FRAGMENT OF RECOMBINANT HUMAN FIBRONECTIN IN THE PRESENCE OF MEDIUM CONTAINING STEM CELL FACTOR (SCF), FMS-RELATED TYROSINE KINASE 3 LIGAND (FLT-3L), THROMBOPOIETIN (TPO), INTERLEUKIN 3 (IL-3))...;PROPOSED INN: LIST 125, RECOMMENDED INN: LIST 87