(INNC)
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS (HSC), OBTAINED BY LEUKAPHERESIS OR FROM BONE MARROW, TRANSDUCED EX VIVO WITH A NON-REPLICATING, SELFINACTIVATING (SIN) LENTIVIRAL VECTOR ENCODING CODON OPTIMISED HUMAN ADENOSINE DEAMINASE (ADA) UNDER THE INTERNAL CONTROL OF ELONGATION FACTOR 1ALPHA SHORT PROMOTER (EFS)(WHO)
SIMOLADAGENUM AUTOTEMCELUM(INN)