The DOI matches its Crossref record; 87 of the 87 references it cites were found in a registry or an index.
Concise Review: Lessons Learned From Clinical Trials of Gene Therapy in Monogenic Immunodeficiency Diseases
Abstract
Thirty years ago, retroviral transfer of genetic material into hematopoietic stem and progenitor cells (HSC/Ps) led to predictions that this technology would transform modern medicine [Nature 1983;305:556–558; Nature 1984;310:476–480]. Studies in several immunodeficiency diseases in the past 15 years have demonstrated clear proof of principle that gene therapy can have long-lasting, potentially curative effects without the need to search for allogeneic donors and without risk of graft-versus-host disease. Improvement in gene transfer efficiency for target HSC/Ps brought to light issues of insertional mutagenesis caused by transfer vectors, resulting in oncogene transactivation and leukemias. Lessons from these adverse events have now led to a new generation of vectors, refinements in conditioning regimens, and manufacturing, which are paving the way for expanded applications of the current technology and recent emphasis on gene targeting/genome editing as the next advancements in the field.
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“Gene therapy with autologous hematopoietic stem cells has been investigated as an alternative to allogeneic hematopoietic stem-cell transplantation.11–13”
From Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy (2017) · cites this paper as [13]
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Cites
LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science · doi:10.1126/science.1088547 · Cited by 3,587
Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.-L., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,167
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Hacein-Bey-Abina, S., Garrigue, A., Wang, G. P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35700 · Cited by 1,854
Chronic Granulomatous Disease: Report on a National Registry of 368 Patients
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T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T. A., Clerici, M., Shearer, G. M., Chang, L., Chiang, Y. L., Tolstoshev, P., et al. (1995) · Science · doi:10.1126/science.270.5235.475 · Cited by 1,584
Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration
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Howe, S. J., Mansour, M. R., Schwarzwaelder, K., Bartholomae, C., Hubank, M., Kempski, H., Brugman, M. H., Pike-Overzet, K., Chatters, S. J., de Ridder, D., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35798 · Cited by 1,286
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
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Self-citationLentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science · doi:10.1126/science.1233151 · Cited by 1,025
Cited by
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-citationLineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
Brendel, C., Guda, S., Renella, R., Bauer, D. E., Canver, M. C., Kim, Y.-J., Heeney, M. M., Klatt, D., Fogel, J., Milsom, M. D., et al. (2016) · Journal of Clinical Investigation · doi:10.1172/jci87885 · Cited by 167
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Metadata
- Authors
- Williams, D. A., & Thrasher, A. J.
- Affiliations
- Division of Hematology/Oncology, Boston Children's Hospital, and Department of Pediatric Oncology, Dana Farber Cancer Institute, Harvard Medical School, Harvard Stem Cell Institute, Boston, Massachusetts, USA
- Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, London, United Kingdom
- Venue
- Stem Cells Translational Medicine
- Publisher
- Oxford University Press (OUP)
- Published
- 2014-03-28
- Volume
- 3
- Issue
- 5
- Pages
- 636-642
- ISSN
- 2157-6564
- 2157-6580
- Cited by
- 56
- References
- 87
- Language
- en
- Licence
- academic.oup.com/journals/pages/open_access/funder_policies/chorus/standard_publication_model (vor)
- Funders
- Wellcome Trust
- National Institute of Health Research
- Great Ormond Street Hospital National Health Service Trust Biomedical Research Center
- Medical Research Council (U01A1087628, U01HL117720, HLBII-S-11-001403)
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- Williams, D. A., & Thrasher, A. J. (2014). Concise Review: Lessons Learned From Clinical Trials of Gene Therapy in Monogenic Immunodeficiency Diseases. Stem Cells Translational Medicine. https://doi.org/10.5966/sctm.2013-0206
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
