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

Autologous CD34+ hematopoietic stem cells, isolated from childhood cerebral adrenoleukodystrophy (CCALD) patients and transduced ex-vivo with the Lenti-D lentiviral vector, to achieve expression of a functioning copy of the ABCD-1 gene.

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. 

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Adult Stem / Progenitor Cell
Homo sapiens
ABCD-1-transduced peripheral blood-derived hematopoietic stem cells