Firolimogene autotemcel
firolimogene autotemcel
AUTOLOGOUS CD34+ CELLS ISOLATED FROM BONE MARROW OF NEWLY DIAGNOSED X-LINKED SEVERE COMBINED IMMUNODEFICIENCY (X-SCID) PATIENTS, TRANSDUCED EX VIVO WITH A SELF-INACTIVATING LENTIVIRAL VECTOR, ENCODING CODON-OPTIMIZED HUMAN INTERLEUKIN-2 RECEPTOR SUBUNIT
autologous cd34+ cells isolated from bone marrow of newly diagnosed x-linked severe combined immunodeficiency (x-scid) patients, transduced ex vivo with a self-inactivating lentiviral vector, encoding codon-optimized human interleukin-2 receptor subunit