EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
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ABCD-1-transduced hematopoietic stem cells for the treatment of childhood cerebral X-linked adrenoleukodystrophy (CCALD)

This clinical study will assess the efficacy and safety of autologous CD34+ hematopoietic stem cells (HSCs), transduced ex-vivo with the Lenti-D-bearing lentiviral vector, in the treatment of childhood cerebral adrenoleukodystrophy (CCALD). Modified stem cells will then be re-infused back into the subject following myeloablative conditioning. The first expected outcome is ALD protein expression in granulocytes, monocytes, and T and B lymphocytes, followed by cerebral demyelination.

Regimen of delivery is based on a French pilot study that included a gene therapy trial in two ALD patients. Autologous CD34+ cells were removed from the patients, genetically corrected ex vivo with a lentiviral vector encoding wild-type ABCD1, and then re-infused into the patients after they had received myeloablative treatment. 14 to 16 months after infusion, progressive cerebral demyelination in the two patients halted. The study was sponsored by INSERM, licensed by Bluebird Bio and reported in the November 2009 issue of Science.
ABCD-1-transduced hematopoietic stem cells for the treatment of childhood cerebral X-linked adrenoleukodystrophy (CCALD)
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