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Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency
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“Subsequently, investigators in the UK and the United States, developed a lentiviral vector (LVV) for ADA SCID gene therapy which is a SIN vector lacking long-term repeat (LTR) enhancers and drives transcription of a codon-optimized ADA cDNA from the elongation factor α 1 short (EFS) promotor, which has minimal genotoxic potential.26”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [26]
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In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
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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
Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration
Wu, X., Li, Y., Crise, B., & Burgess, S. M. (2003) · Science · doi:10.1126/science.1083413 · Cited by 1,305
Transfusion 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
Self-citationMontini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37630 · Cited by 571
Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors
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Cited by
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
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy
Williams, D. A., Kohn, D. B., & Thrasher, A. J. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.058 · Cited by 11
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Metadata
- Authors
- Carbonaro, D. A., Zhang, L., Jin, X., Montiel-Equihua, C., Geiger, S., Carmo, M., Cooper, A., Fairbanks, L., Kaufman, M. L., Sebire, N. J., Hollis, R. P., Blundell, M. P., Senadheera, S., Fu, P.-Y., Sahaghian, A., Chan, R. Y., Wang, X., Cornetta, K., Thrasher, A. J., Kohn, D. B., & Gaspar, H. B.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2014-03
- Volume
- 22
- Issue
- 3
- Pages
- 607-622
- ISSN
- 1525-0016
- Cited by
- 78
- References
- 6
- Language
- en
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