Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

By N. Cartier, S. Hacein-Bey-Abina, C. Bartholomae, G. Veres, M. Schmidt, Ina Kutschera, M. Vidaud, U. Abel, L. Dal-Cortivo, L. Caccavelli and 16 more. Science, 2009

1,461 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/a8a48a8569a8f49df9106be68a42eaee94b36250

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In Genetics

1. M. Kay
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Citations in the corpus

• State-of-the-art gene-based therapies: the road ahead (2011, background): “Conversely, no predominant clones containing the integrated lentiviral provirus were seen (after a 2 to 4 year follow-up) in transduced haematopoietic cells in patients who were treated for X-linked adrenoleukodystrophy (X-ALD”
• Clinical use of lentiviral vectors (2018, background): “Lentiviral vector-based gene transfer into CD34+HSCs has subsequently been applied in the treatment of several genetic diseases, including β-thalassemia [67], X-linked adrenoleukodystrophy [51], metachromatic leukodystrophy [68, 69], and Wiskott-Aldrich Syndrome [70].”
• Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy (2013, methodology): “These gene classes were the same, or shared >75% genes, with gene classes overrepresented in the ALD HSC-GT (17, 29) (Fig.”
• Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo (2010, neutral): “…in gene therapy for patients with genetic diseases such as ADA-SCID (59–60), chronic granulomatous disease (61) and X-linked adrenoleukodystrophy (62), is that non-myeloablative conditioning can be used to facilitate engraftment of gene-modified autologous HSC with minimal associated toxicity.”
• Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease (2010, background): “Improved vector designs should enable the safe and effective application of gene therapy for the long-term correction of CGD and other inherited disorders, as demonstrated recentl”
• Gene therapy clinical trials worldwide to 2017: An update (2018, background): “the movie Lorenzo's Oil, this was the first report of a lentiviral vector being used successfully to treat a genetic disease in humans, as well as the first time that a severe disease of the central nervous system had been treated with measurable efficacy.(15) There were also reports of the first su”