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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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Ott, M., Stein, S., Schultze‐Strasser, S., Jauch, A. S., Burwinkel, B., Schlegelberger, B., Goehring, G., Preiss, C., Kramer, R., Schwarzwaelder, K., et al. (2007) · Blood · doi:10.1182/blood.v110.11.503.503 · Cited by 3
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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
Self-citation
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)
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
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.
