(INNC)
AUTOLOGOUS CD34+ CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD, TRANSDUCED WITH A SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN ALPHA-L-IDURONIDASE (IDUA) 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 HIV-1 PSI PACKAGING SIGNAL, A REV RESPONSE ELEMENT (RRE), A CENTRAL POLYPURINE TRACT (CPPT) SEQUENCE AND THE WOODCHUCK HEPATITIS VIRUS POST-TRANSCRIPTIONAL REGULATORY ELEMENT (WPRE). THE VECTOR IS PSEUDOTYPED WITH THE ENVELOPE GLYCOPROTEIN (G) OF THE VESICULAR STOMATITIS VIRUS (VSV). THE CD34+ CELL POPULATION WAS ENRICHED USING IMMUNOMAGNETIC MICROBEADS, AND THE CELLS CULTURED IN THE PRESENCE OF MEDIA CONTAINING FMSRELATED TYROSINE KINASE 3 LIGAND (FLT-3L), INTERLEUKIN 3 (IL-3), THROMBOPOIETIN (TPO) AND STEM CELL FACTOR (SCF). THE FINAL SUBSTANCE CONTAINS MORE OR EQUAL TO 70% CD34+ CELLS; REC. INN: LIST 87(WHO)
AZAMIDUGENUM AUTOTEMCELUM(INN)