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A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
Abstract
To the Editor: We recently reported (April 18 issue)1 the sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease. These results have since been confirmed in four additional patients with typical X-linked severe combined immunodeficiency. Of the first four successfully treated patients, three continue to do well up to 3.6 years after gene therapy, whereas a serious adverse event occurred in the fourth patient. At a routine checkup 30 months after gene therapy, lymphocytosis consisting of a monoclonal population . . .
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“Lentiviral vectors are considered safer than gamma-retroviral vectors in terms of their semi-random integration patterns, as gene integration does not preferentially occur in start regions of active genes (93-95).”
From Scalable Manufacturing of CAR T Cells for Cancer Immunotherapy (2021) · cites this paper as [93]
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“Unfortunately, despite the successful clinical benefit achieved with that γ-retrovirus therapy, multiple patients developed leukemia in that trial due to insertional mutagenesis [145, 146].”
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“Gelsinger and cases of leukemia after treating X-linked severe combined immunodeficiency using adenoviruses (though Alain Fischer was awarded the well-earned Japan Prize last year) [54,55].”
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Thomas, S., Klobuch, S., Besold, K., Plachter, B., Dörrie, J., Schaft, N., Theobald, M., & Herr, W. (2012) · European Journal of Immunology · doi:10.1002/eji.201242666 · Cited by 17
Suttiprapa, S., Rinaldi, G., & Brindley, P. (2011) · Transgenic research · doi:10.1007/s11248-011-9556-0 · Cited by 17
“…5 0 -LTRs. Interest in retroviral vectors also is tempered, particularly in clinical settings, by issues of genotoxicity involving the activation of cellular oncogenes flanking sites of vector integration (Bohne and Cathomen 2008; Hacein-Bey-Abina et al. 2003; Li et al. 2009; Ott et al. 2006).”
Gene Therapy - Developments and Future Perspectives
Kang, C. (2011) · InTech eBooks · doi:10.5772/748 · Cited by 16
Conditionally replicating lentiviral-hybrid episomal vectors for suicide gene therapy.
Vargas, J. J., Klotman, M., & Cara, A. (2008) · Antiviral Research · doi:10.1016/j.antiviral.2008.06.015 · Cited by 17
Perinatal stem-cell and gene therapy for hemoglobinopathies.
Surbek, D., Schoeberlein, A., & Wagner, A.-M. (2008) · Seminars in Fetal & Neonatal Medicine · doi:10.1016/j.siny.2008.03.002 · Cited by 17
Kim, J. H., Park, S.-N., & Suh, H. (2007) · Yonsei medical journal · doi:10.3349/ymj.2007.48.1.109 · Cited by 17
Translational considerations for CNS gene therapy
Ryan, D. A., & Federoff, H. J. (2007) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.7.3.305 · Cited by 16
“Cancer is one result of integration near an oncogene, as was originally thought in the X-linked severe combined immunodeficiency gene therapy trial [2].”
Background
Metadata
- Authors
- Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.-L., Fraser, C. C., Cavazzana-Calvo, M., & Fischer, A.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2003-01-16
- Volume
- 348
- Issue
- 3
- Pages
- 255-256
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W2323613689
- Cited by
- 1,867
- References
- 0
- Citations by year
- 2026: 29 · 2025: 32 · 2024: 31 · 2023: 25 · 2022: 47 · 2021: 52 · 2020: 46 · 2019: 50 · 2018: 45 · 2017: 63 · 2016: 65 · 2015: 84 · 2014: 87 · 2013: 92 · 2012: 92
- FWCI
- 86.98
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- FreiDok plus (Universitätsbibliothek Freiburg) (submittedVersion)
- DigitalGeorgetown (Georgetown University Library) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Immunology and Microbiology › Immunology › Immunodeficiency and Autoimmune Disorders
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Keywords
- X-linked severe combined immunodeficiency, gene therapy, adverse events, gene therapy safety
- MeSH terms
- Chromosomes, Human, Pair 11, DNA-Binding Proteins, Humans, Genetic Linkage, Metalloproteins, Proto-Oncogene Proteins, Retroviridae, Genetic Therapy, Gene Expression, Mutagenesis, Insertional, Severe Combined Immunodeficiency, Lymphocyte Count, Chromosomes, Human, X, Adaptor Proteins, Signal Transducing, Precursor Cell Lymphoblastic Leukemia-Lymphoma, LIM Domain Proteins
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