Cited by 1,867Virus-based gene therapy researchImmunodeficiency and Autoimmune DisordersCAR-T cell therapy research

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A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, Salima, von Kalle, Christof, Schmidt, Manfred, Le Deist, Françoise, Wulffraat, Nicolas, McIntyre, Elisabeth, Radford, Isabelle, Villeval, Jean-Luc, Fraser, Christopher C., Cavazzana-Calvo, Marina, Fischer, Alain·2003·New England Journal of Medicine

DOI 10.1056/nejm200301163480314

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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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10.3109/08977199309011023 · DOI
10.1056/NEJM199902183400703 · DOI

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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.
ORCID: Schmidt, Manfred
Villeval, Jean-Luc
Fischer, Alain
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
DOI: 10.1056/nejm200301163480314
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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A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

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