Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
By M. Ott, M. Schmidt, Kerstin Schwarzwaelder, S. Stein, U. Siler, U. Koehl, H. Glimm, K. Kühlcke, A. Schilz, Hana Kunkel and 17 more. Nature Medicine, 2006
1,241 citations recorded by the source corpus.
Subfields: Genetics, Immunology, Oncology
View Paper: https://www.semanticscholar.org/paper/964f58739a2db18af5f330e795ef68abfc42387f
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Citations in the corpus
- Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells (2006, background): “, and retroviral insertion causes only a benign clonal amplification of hematopoietic progenitors in the context of chronic granulomatous diseas”
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy (2013, background): “(HSPCs) has the potential to trigger leukemia through insertionalmutagenesis (14, 18).”
- Gene therapy clinical trials worldwide to 2007—an update (2007, background): “Another monogenic immunodeficiency, chronic granulomatous disease, has also been the target of a successful trial [18].”
- Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo (2010, neutral): “…of autologous HSC transplantation 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…”
- Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease (2010, background): “However, insertional activation of MDS1 – EVI1 and the genes encoding PR domain–containing protein 16 ( PRDM16 ) and SET-binding protein 1 ( SETBP1 ) triggered a threefold to fivefold increase in the number of gene-transduced cells in the peripheral blood of both subject”
- Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration (2006, methodology): “5–5 ng of tumor DNA and 10–100 ng of transduced pretransplant DNA, respectively, as template for LAM-PC”
- State-of-the-art gene-based therapies: the road ahead (2011, background): “However, using a similar vector, non-malignant cellular clonal expansion has been observed in gene therapy trials for chronic granalomatous diseas”
- Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “In April 2006, a report on a Swiss–German phase I/II gene therapy clinical trial aimed at treating chronic granulomatous disease (CGD), an inherited primary immunodeficiency that affects phagocytes, brought new hope of success [39].”