The DOI matches its Crossref record; 5 of the 5 references it cites were found in a registry or an index.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
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“They reported results in 50 treated ADA SCID patients, with 100% survival, and 96% event-free survival, with 2 patients failing to engraft sufficient gene-corrected stem cells for independent immunity.27”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [27]
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“No insertional-related events have been observed out to 6-11 years from treatment, with stable persistence of protective immunity.”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [27]
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Cites
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508
Self-citationGene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1705342 · Cited by 684
Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1700554 · Cited by 604
Self-citationSessa, M., Lorioli, L., Fumagalli, F., Acquati, S., Redaelli, D., Baldoli, C., Canale, S., Lopez, I. D., Morena, F., Calabria, A., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30374-9 · Cited by 502
Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors
Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., & Baum, C. (2008) · Molecular Therapy · doi:10.1038/mt.2008.5 · Cited by 271
Research works printed but not linked: 28
- 10.1016/S0090-1229(95)90288-0 · DOI
- 10.3389/fimmu.2012.00265 · DOI
- Hershfield MS. Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy. Semin Hematol 1998;35:291-298.9801258
- 10.1016/j.jaci.2018.08.024 · DOI
- Booth C, Gaspar HB. Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics 2009;3:349-358.19707420
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- Haddad E, Landais P, Friedrich W, et al. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood 1998;91:3646-3653.9573000
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- Kohn DB, Shaw KL, Garabedian E, et al. Lentiviral gene therapy with autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID): two year follow-up results. Mol Ther 2020;28:Suppl 1:1300-1300. abstract.
- Kuo CY, Puck JM, Logan BR, et al. Adenosine deaminase (ADA)-deficient severe combined immune deficiency (SCID): analysis of cases enrolled in protocols of the Primary Immune Deficiency Treatment Consortium (PIDTC). J Clin Immunol 2018;38:342-343. abstract.
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Cited by
Engineered stem cells by emerging biomedical stratagems
Wang, J., Zhang, X., Chen, H., Ren, H., Zhou, M., & Zhao, Y. (2024) · Science Bulletin · doi:10.1016/j.scib.2023.12.006 · Cited by 37
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy
Williams, D. A., Kohn, D. B., & Thrasher, A. J. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.058 · Cited by 11
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Metadata
- Authors
- Kohn, D. B., Booth, C., Shaw, K. L., Xu-Bayford, J., Garabedian, E., Trevisan, V., Carbonaro-Sarracino, D. A., Soni, K., Terrazas, D., Snell, K., Ikeda, A., Leon-Rico, D., Moore, T. B., Buckland, K. F., Shah, A. J., Gilmour, K. C., De Oliveira, S., Rivat, C., Crooks, G. M., Izotova, N., Tse, J., Adams, S., Shupien, S., Ricketts, H., Davila, A., Uzowuru, C., Icreverzi, A., Barman, P., Campo Fernandez, B., Hollis, R. P., Coronel, M., Yu, A., Chun, K. M., Casas, C. E., Zhang, R., Arduini, S., Lynn, F., Kudari, M., Spezzi, A., Zahn, M., Heimke, R., Labik, I., Parrott, R., Buckley, R. H., Reeves, L., Cornetta, K., Sokolic, R., Hershfield, M., Schmidt, M., Candotti, F., Malech, H. L., Thrasher, A. J., & Gaspar, H. B.
- Affiliations
- From the Departments of Microbiology, Immunology, and Molecular Genetics (D.B.K., K.L.S., D.A.C.-S., D.T., A.D., A. Icreverzi, P.B., B.C.F., R.P.H., M.C., A.Y., K.M.C., C.E.C., R.Z.), Pediatrics (D.B.K., T.B.M., S.D.O., S.S.), and Pathology and Laboratory Medicine (G.M.C.) and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (D.B.K., G.M.C.), University of California, Los Angeles (UCLA), and the Department of Pharmaceutical Services, Ronald Reagan UCLA Medical Center (J.T.), Los Angeles, and Stanford School of Medicine, Palo Alto (A.J.S.) — all in California; University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust (C.B., J.X.-B., V.T., K. Soni, K. Snell, D.L.-R., K.F.B., K.C.G., C.R., N.I., S.A., H.R., C.U., A.J.T., H.B.G.), and Orchard Therapeutics (Europe) (D.A.C.-S., S.A., F.L., M.K., A.S., H.B.G.) — all in London; the National Institute of Allergy and Infectious Diseases (H.L.M.) and the Na...
- ORCID
- Candotti, Fabio
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2021-05-27
- Volume
- 384
- Issue
- 21
- Pages
- 2002-2013
- ISSN
- 0028-4793
- 1533-4406
- PMC
- PMC8240285
- Cited by
- 217
- References
- 5
- Language
- en
- Funders
- National Gene Vector Biorepository (NHLBI Contract No. 75N92019D00018)
- California Institute for Regenerative Medicine (AC1-07675, CL1-00505, CLIN2-09339, FA1-00613)
- Medical Research Council (G80248, MR/K015427/1)
- National Institute for Health Research Biomedical Research Centre at the Great Ormond Street Hospital for Children NHS Foundation Trust and University College London (AC1-07675)
- Orchard Therapeutics Ltd (NA)
- National Institutes of Health (2P01 HL073104, U01 AI100801)
- National Heart, Lung, and Blood Institute (Gene Therapy Resource Program [GTRP] Request for S)
- National Human Genome Research Institute (Intramural program)
- National Institute of Allergy and Infectious Diseases (Intramural program)
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