ABCD-1-transduced peripheral blood-derived hematopoietic stem cells (bluebird bio)

Peripheral blood mononuclear cells (PB-MNCs) were collected from patients after stimulation by intravenous injection of G-CSF. After selecting CD34⁺cells using an immunomagnetic  selection procedure, cells were pre-activated ex vivo with a cytokine mixture. The cells were then infected with a replication-defective HIV-1–derived lentiviral vector (CG1711 hALD) expressing wild-type ABCD1 cDNA under the control of the MND promoter. Transduced CD34⁺ cells were cryopreserved until re-infusion into CCALD patients.