Myelodysplasia after Lentiviral Gene Therapy

Abstract excerpt

To the Editor: Duncan et al. (Oct. 10 issue) 1 report the occurrence of myelodysplastic syndrome in approximately 10% of patients with cerebral adrenoleukodystrophy who received autologous hematopoietic stem-cell gene therapy with a lentiviral vector. The design of this lentiviral vector raises unique safety risks, because expression of the therapeutic transgene is driven by a potent murine γ-retroviral...

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