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Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
Abstract
More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID-X1 clinical studies, based on first-generation gammaretroviral vectors, demonstrated good long-term immune reconstitution in most treated patients despite the occurrence of vector-related leukemia in a few of them. This gene therapy has successfully enabled correction of the T cell defect. Natural killer and B cell defects were only partially restored, most likely due to the absence of a conditioning regimen. The success of these pioneering trials paved the way for the extension of gene-based treatment to many other diseases of the hematopoietic system, but the unfortunate serious adverse events led to extensive investigations to define the retrovirus integration profiles. This review puts into perspective the clinical experience of gene therapy for SCID-X1, with the development and implementation of new generations of safer vectors such as self-inactivating gammaretroviral or lentiviral vectors as well as major advances in integrome knowledge.
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“Of the 20 patents participating in the trial, 17 were successfully and stably cured (Cavazzana et al., 2016).”
From Delivering CRISPR: a review of the challenges and approaches (2018) · cites this paper as Cavazzana et al., 2016
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“Self-inactivating vectors have formally demonstrated their improved safety profile (relative to LTR-driven retroviral vectors), as no SAEs have been observed since their first use in clinical trials in 2006.(71) Another dogma has been also deeply challenged by the last 20 years of RIS analysis: changes due to the clon…”
Self-citationTransfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
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Delivering CRISPR: a review of the challenges and approaches
Lino, C. A., Harper, J. C., Carney, J. P., & Timlin, J. A. (2018) · Drug Delivery · doi:10.1080/10717544.2018.1474964 · Cited by 1,185
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The challenge and prospect of mRNA therapeutics landscape
Weng, Y., Li, C., Yang, T., Hu, B., Zhang, M., Guo, S., Xiao, H., Liang, X.-J., & Huang, Y. (2020) · Biotechnology Advances · doi:10.1016/j.biotechadv.2020.107534 · Cited by 417
Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease
Marquez Loza, L. I., Yuen, E. C., & McCray, P. B. (2019) · Genes · doi:10.3390/genes10030218 · Cited by 59
Metadata
- Authors
- Cavazzana, M., Six, E., Lagresle-Peyrou, C., André-Schmutz, I., & Hacein-Bey-Abina, S.
- Affiliations
- Necker Children's Hospital
- Groupe Hospitalier Universitaire Ouest
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2016-01-20
- Volume
- 27
- Issue
- 2
- Pages
- 108-116
- ISSN
- 1043-0342
- 1557-7422
- Cited by
- 106
- References
- 11
- Language
- en
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