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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
Abstract
Lentiviral gene therapy with conditioning achieves humoral reconstitution in older SCID-X1 patients.
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“In studies at St. Jude Children's Cancer Research Hospital, at the National Institute of Allergy and Infectious Diseases at the NIH, and in ongoing collaborative trials in the UK and the United States, more than 30 XSCID patients have been treated, including older patients who had prior allogeneic HSCT with poor immune reconstitution.”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [29]
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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
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., et al. (2003) · New England Journal of Medicine · doi:10.1056/nejm200301163480314 · Cited by 1,867 · cited 4×
“A possible explanation for the relative survival advantage of autologous gene-corrected T cells may be the better crosstalk between autologous T cells and the other immune cells or the constitutive expression of gc, although no increases in the expression of gc or its downstream signaling component JAK3 (Janus kinase…”
BackgroundInfluentialTranscription Start Regions in the Human Genome Are Favored Targets for MLV Integration
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Self-citationTransfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature · doi:10.1038/nature09328 · Cited by 1,276
Specific HIV integration sites are linked to clonal expansion and persistence of infected cells
Maldarelli, F., Wu, X., Su, L., Simonetti, F. R., Shao, W., Hill, S., Spindler, J., Ferris, A. L., Mellors, J. W., Kearney, M. F., et al. (2014) · Science · doi:10.1126/science.1254194 · Cited by 849
Self-citationA Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
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., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1404588 · Cited by 412
Self-citationEnhancers Are Major Targets for Murine Leukemia Virus Vector Integration
De Ravin, S. S., Su, L., Theobald, N., Choi, U., Macpherson, J. L., Poidinger, M., Symonds, G., Pond, S. M., Ferris, A. L., Hughes, S. H., et al. (2014) · Journal of Virology · doi:10.1128/jvi.00011-14 · Cited by 103
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Cited by
Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science · doi:10.1126/science.aan4672 · Cited by 1,410
Gene therapy clinical trials worldwide to 2017: An update
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3015 · Cited by 903
High, K. A., & Roncarolo, M. G. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1706910 · Cited by 555
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Tambuyzer, E., Vandendriessche, B., Austin, C. P., Brooks, P. J., Larsson, K., Miller Needleman, K. I., Valentine, J., Davies, K., Groft, S. C., Preti, R., et al. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0049-9 · Cited by 357
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
Mamcarz, E., Zhou, S., Lockey, T., Abdelsamed, H., Cross, S. J., Kang, G., Ma, Z., Condori, J., Dowdy, J., Triplett, B., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1815408 · Cited by 263
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Metadata
- Authors
- De Ravin, S. S., Wu, X., Moir, S., Kardava, L., Anaya-O’Brien, S., Kwatemaa, N., Littel, P., Theobald, N., Choi, U., Su, L., Marquesen, M., Hilligoss, D., Lee, J., Buckner, C. M., Zarember, K. A., O’Connor, G., McVicar, D., Kuhns, D., Throm, R. E., Zhou, S., Notarangelo, L. D., Hanson, I. C., Cowan, M. J., Kang, E., Hadigan, C., Meagher, M., Gray, J. T., Sorrentino, B. P., & Malech, H. L.
- Affiliations
- Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
- Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
- Laboratory of Immunoregulation, NIAID, NIH, Bethesda, MD 20892, USA.
- Cancer and Inflammation Program, National Cancer Institute Frederick, Frederick, MD 21702, USA.
- Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN 38105, USA.
- Division of Immunology, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA.
- Texas Children’s Hospital, Houston, TX 77030, USA.
- Department of Pediatrics, Benioff Children’s Hospital, and University of California, San Francisco, San Francisco, CA, USA.
- Audentes Therapeutics, San Francisco, CA 94101, USA.
- Venue
- Science Translational Medicine
- Published
- 2016-04-20
- Volume
- 8
- Issue
- 335
- ISSN
- 1946-6234
- 1946-6242
- PMC
- PMC5557273
- Cited by
- 244
- References
- 6
- Language
- en
- Funders
- Intramural Research Program of NIAID
- National Heart, Lung, and Blood Institute (P01HL 53749)
- NIH-NIAID (U54 AI 082973)
- Frederick National Laboratory for Cancer Research
- NIH (Z01-A1-00644, Z01-A1-00988, HHSN261200800001E)
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