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Cartier et al. report another major advanceâthe successful first clinical testing of an HIV-derived vector in hematopoietic stem cell (HSC)âbased gene therapy. The procedure was used to treat a severe neurodegenerative disease, X-linked adrenoleukodystrophy (ALD), and the results indicate stable expression of a therapeutic gene in a substantial fraction of patients' hematopoietic cells, as well as clinical benefits.
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