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Cited by 248

The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.

Lentiviral gene therapy for X-linked chronic granulomatous disease

the Net4CGD consortium, Kohn, Donald B., Booth, Claire, Kang, Elizabeth M., Pai, Sung-Yun, Shaw, Kit L., Santilli, Giorgia, Armant, Myriam, Buckland, Karen F., Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J., Kuo, Caroline Y., Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C., Terrazas, Dayna, Wang, Leo D., Bauser, Christopher A., Paprotka, Tobias, Kuhns, Douglas B., Gregg, John, Raymond, Hayley E., Everett, John K., Honnet, Geraldine, Biasco, Luca, Newburger, Peter E., Bushman, Frederic D., Grez, Manuel, Gaspar, H. Bobby, Williams, David A., Malech, Harry L., Galy, Anne, Thrasher, Adrian J.·2020·Nature Medicine
DOI 10.1038/s41591-019-0735-5PMC PMC7115833
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Research works printed but not linked: 29

  1. Winkelstein, J. A. et al. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore) 79, 155–169 (2000). · DOI · Paper page
  2. Holland, S. M. Chronic granulomatous disease. Hematol. Oncol. Clin. N. Am. 27, 89–99 (2013). · DOI
  3. Gungor, T. et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet 383, 436–448 (2014). · DOI
  4. Parta, M. et al. Allogeneic reduced-intensity hematopoietic stem cell transplantation for chronic granulomatous disease: a single-center prospective trial. J. Clin. Immunol. 37, 548–558 (2017). · DOI
  5. Ott, M. G. et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401–409 (2006). · DOI · Paper page
  6. Kang, E. M. et al. Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 115, 783–791 (2010). · DOI
  7. Stein, S. et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 16, 198–204 (2010). · DOI · Paper page
  8. Santilli, G. et al. Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells. Mol. Ther. 19, 122–132 (2011). · DOI · Paper page
  9. Brendel, C. et al. Non-clinical efficacy and safety studies on G1XCGD, a lentiviral vector for ex vivo gene therapy of X-linked chronic granulomatous disease. Hum. Gene Ther. Clin. Dev. 29, 69–79 (2018). · DOI
  10. Schroder, A. R. et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521–529 (2002). · DOI · Paper page
  11. Berry, C. C. et al. Estimating abundances of retroviral insertion sites from DNA fragment length data. Bioinformatics 28, 755–762 (2012). · DOI
  12. Dulamea, A. O. & Lupescu, I. G. Neurological complications of hematopoietic cell transplantation in children and adults. Neural Regen. Res. 13, 945–954 (2018). · DOI
  13. Simonis, A. et al. Allogeneic hematopoietic cell transplantation in patients with GATA2 deficiency-a case report and comprehensive review of the literature. Ann. Hematol. 97, 1961–1973 (2018). · DOI
  14. Weisser, M. et al. Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions. J. Allergy Clin. Immunol. 138, 219–228 e219 (2016). · DOI · Paper page
  15. Grez, M. et al. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol. Ther. 19, 28–35 (2011). · DOI
  16. Dinauer, M. C., Gifford, M. A., Pech, N., Li, L. L. & Emshwiller, P. Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease. Blood 97, 3738–3745 (2001). · DOI
  17. Marciano, B. E. et al. X-linked carriers of chronic granulomatous disease: illness, lyonization, and stability. J. Allergy Clin. Immunol. 141, 365–371 (2018). · DOI
  18. Zanta-Boussif, M. A. et al. Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. Gene Ther. 16, 605–619 (2009). · DOI
  19. Merten, O. W. et al. Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum. Gene Ther. 22, 343–356 (2011). · DOI
  20. Krueger, F. & Andrews, S. R. Bismark: a flexible aligner and methylation caller for Bisulfite-Seq applications. Bioinformatics 27, 1571–1572 (2011). · DOI
  21. Langmead, B. et al. Ultrafast and memory-efficient alignment of short DNA sequences to the human genome. Genome Biol. 10, R25 (2009). · DOI · Paper page
  22. Liu, Y. et al. Bis-SNP: combined DNA methylation and SNP calling for Bisulfite-Seq data. Genome Biol. 13, R61 (2012). · DOI
  23. McKenna, A. et al. The genome analysis toolkit: a MapReduce framework for analyzing next-generation DNA sequencing data. Genome Res. 20, 1297–1303 (2010). · DOI · Paper page
  24. DePristo, M. A. et al. A framework for variation discovery and genotyping using next-generation DNA sequencing data. Nat. Genet. 43, 491–498 (2011). · DOI
  25. Sherman, E. et al. INSPIIRED: a pipeline for quantitative analysis of sites of new DNA integration in cellular genomes. Mol. Ther. Methods Clin. Dev. 4, 39–49 (2017). · DOI
  26. Berry, C. C. et al. INSPIIRED: quantification and visualization tools for analyzing integration site distributions. Mol. Ther. Methods Clin. Dev. 4, 17–26 (2017). · DOI
  27. Berry, C. et al. Selection of target sites for mobile DNA integration in the human genome. PLoS Comput. Biol. 2, e157 (2006). · DOI
  28. Biasco, L. et al. In vivo tracking of human hematopoiesis reveals patterns of clonal dynamics during early and steady-state reconstitution phases. Cell Stem Cell 19, 107–119 (2016). · DOI · Paper page
  29. Scala, S. et al. Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans. Nat. Med. 24, 1683–1690 (2018). · DOI

Cited by

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Metadata

Authors
the Net4CGD consortium, Kohn, D. B., Booth, C., Kang, E. M., Pai, S.-Y., Shaw, K. L., Santilli, G., Armant, M., Buckland, K. F., Choi, U., De Ravin, S. S., Dorsey, M. J., Kuo, C. Y., Leon-Rico, D., Rivat, C., Izotova, N., Gilmour, K., Snell, K., Dip, J. X.-B., Darwish, J., Morris, E. C., Terrazas, D., Wang, L. D., Bauser, C. A., Paprotka, T., Kuhns, D. B., Gregg, J., Raymond, H. E., Everett, J. K., Honnet, G., Biasco, L., Newburger, P. E., Bushman, F. D., Grez, M., Gaspar, H. B., Williams, D. A., Malech, H. L., Galy, A., & Thrasher, A. J.
ORCID
Venue
Nature Medicine
Publisher
Springer Science and Business Media LLC
Published
2020-01-27
Volume
26
Issue
2
Pages
200-206
ISSN
  • 1078-8956
  • 1546-170X
DOI
10.1038/s41591-019-0735-5
PMC
PMC7115833
Cited by
248
References
4
Language
en
Licence
Funders
  • Borchard Foundation Center on Law and Aging
  • Orchard Therapeutics

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CoChat. (2026). Citation check: Lentiviral gene therapy for X-linked chronic granulomatous disease. https://app.cochat.ai/papers/10.1038/s41591-019-0735-5
The paper
the Net4CGD consortium, Kohn, D. B., Booth, C., Kang, E. M., Pai, S.-Y., Shaw, K. L., Santilli, G., Armant, M., Buckland, K. F., Choi, U., De Ravin, S. S., Dorsey, M. J., Kuo, C. Y., Leon-Rico, D., Rivat, C., Izotova, N., Gilmour, K., Snell, K., Dip, J. X.-B., . . . Thrasher, A. J. (2020). Lentiviral gene therapy for X-linked chronic granulomatous disease. Nature Medicine. https://doi.org/10.1038/s41591-019-0735-5