A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
Open accessAbstract
BACKGROUND: In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted in vector-induced leukemia through enhancer-mediated mutagenesis in 25% of patients. We assessed the efficacy and safety of a self-inactivating retrovirus for the treatment of SCID-X1. METHODS: We enrolled nine boys with SCID-X1 in parallel trials in Europe and the United States to evaluate treatment with a self-inactivating (SIN) γ-retrovirus vector containing deletions in viral enhancer sequences expressing γc (SIN-γc). RESULTS: All patients received bone marrow-derived CD34+ cells transduced with the SIN-γc vector, without preparative conditioning. After 12.1 to 38.7 months of follow-up, eight of the nine children were still alive. One patient died from an overwhelming adenoviral infection before reconstitution with genetically modified T cells. Of the remaining eight patients, seven had recovery of peripheral-blood T cells that were functional and led to resolution of infections. The patients remained healthy thereafter. The kinetics of CD3+ T-cell recovery was not significantly different from that observed in previous trials. Assessment of insertion sites in peripheral blood from patients in the current trial as compared with those in previous trials revealed significantly less clustering of insertion sites within LMO2, MECOM, and other lymphoid proto-oncogenes in our patients. CONCLUSIONS: This modified γ-retrovirus vector was found to retain efficacy in the treatment of SCID-X1. The long-term effect of this therapy on leukemogenesis remains unknown. (Funded by the National Institutes of Health and others; ClinicalTrials.gov numbers, NCT01410019, NCT01175239, and NCT01129544.).
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Self-citation
- Authors
- Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., Caccavelli, L., Cros, G., De Oliveira, S., Fernández, K. S., Guo, D., Harris, C. E., Hopkins, G., Lehmann, L. E., Lim, A., London, W. B., van der Loo, J. C. M., Malani, N., Male, F., Malik, P., Marinovic, M. A., McNicol, A.-M., Moshous, D., Neven, B., Oleastro, M., Picard, C., Ritz, J., Rivat, C., Schambach, A., Shaw, K. L., Sherman, E. A., Silberstein, L. E., Six, E., Touzot, F., Tsytsykova, A., Xu-Bayford, J., Baum, C., Bushman, F. D., Fischer, A., Kohn, D. B., Filipovich, A. H., Notarangelo, L. D., Cavazzana, M., Williams, D. A., & Thrasher, A. J.
- Affiliations
- Boston Children's Hospital (US)
- Great Ormond Street Hospital (GB)
- Cincinnati Children's Hospital Medical Center (US)
- Inserm (FR)
- University College London (GB)
- Peoria campus of the University of Illinois System (US)
- Institut Pasteur (FR)
- University of Pennsylvania (US)
- Clinica Santa Maria (CL)
- Sorbonne Paris Cité (FR)
- Garrahan Hospital (AR)
- Medizinische Hochschule Hannover (DE)
- University of California, Los Angeles (US)
- Harvard University (US)
- ORCID
- Pai, Sung-Yun
- Gaspar, H. Bobby
- Armant, Myriam
- Bleesing, Jack
- Buckland, Karen F.
- Caccavelli, Laure
- Cros, Guilhem
- Fernández, Karen S.
- Harris, Chad E.
- Lehmann, Leslie E.
- London, Wendy B.
- Malani, Nirav
- Malik, Punam
- Moshous, Despina
- Ritz, Jerome
- Schambach, Axel
- Shaw, Kit L.
- Sherman, Eric A.
- Silberstein, Leslie E.
- Six, Emmanuelle
- Touzot, Fabien
- Tsytsykova, Alla
- Bushman, Frederic D.
- Fischer, Alain
- Kohn, Donald B.
- Notarangelo, Luigi D.
- Cavazzana, Marina
- Williams, David A.
- Thrasher, Adrian J.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2014-10-09
- Volume
- 371
- Issue
- 15
- Pages
- 1407-1417
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- PMC
- PMC4274995
- OpenAlex
- W2149628283
- Cited by
- 412
- References
- 46
- Citations by year
- 2026: 9 · 2025: 20 · 2024: 17 · 2023: 26 · 2022: 30 · 2021: 17 · 2020: 42 · 2019: 48 · 2018: 36 · 2017: 57 · 2016: 46 · 2015: 56 · 2014: 4 · 2013: 2
- FWCI
- 35.59
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- Wellcome Trust
- National Institute for Health and Care Research (NF-SI-0611-10001)
- National Heart, Lung, and Blood Institute (P01HL073104, HL073104, HHSN268201000009C)
- National Institute of Allergy and Infectious Diseases (U01AI087628, R01AI082020, P30AI045008, AI082020)
- 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, interleukin-2 receptor gamma chain, LMO2, MECOM, gene therapy, CD34+ hematopoietic stem cells, T cell reconstitution, adenovirus infection
- MeSH terms
- Animals, Genetic Vectors, Humans, Infant, Male, Mutation, T-Lymphocytes, Transduction, Genetic, Genetic Therapy, Gene Expression, Gammaretrovirus, DNA, Complementary, Antigens, CD34, Transgenes, Gene Silencing, Mice, Interleukin Receptor Common gamma Subunit, X-Linked Combined Immunodeficiency Diseases
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