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Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque
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“Abnormal dysplastic clonal hematopoiesis was observed in a rhesus macaque that had undergone transplantation with hematopoietic stem and progenitor cells transduced with lentiviral vector containing a strong retroviral murine stem-cell virus constitutive promoter–enhancer in the long terminal repeat sequence.”
From Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy (2024) · cites this paper as [28]
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Cites
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
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508 · cited 5×
“In contrast to the vector utilized in ZL34, the adrenoleukodystrophy trial LV placed the RV promoter and/or enhancer internally instead of within the LV LTR.(4,5,46,47) No clinical trials to date have utilized an LV with an RV promoter and/or enhancer within the LTR.”
MethodInfluentialDunbar, 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
Self-citationTranscription 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
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
Disruption of TET2 promotes the therapeutic efficacy of CD19-targeted T cells
Fraietta, J. A., Nobles, C. L., Sammons, M. A., Lundh, S., Carty, S. A., Reich, T. J., Cogdill, A. P., Morrissette, J. J. D., DeNizio, J. E., Reddy, S., et al. (2018) · Nature · doi:10.1038/s41586-018-0178-z · Cited by 862 · cited 3×
“T cell clone in a patient was linked to an LV insertion inactivating the epigenetic regulator TET2.”
BackgroundGene therapy in a patient with sickle cell disease
Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609677 · Cited by 696
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1705342 · Cited by 684
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 · cited 5×
“In contrast to the vector utilized in ZL34, the adrenoleukodystrophy trial LV placed the RV promoter and/or enhancer internally instead of within the LV LTR.(4,5,46,47) No clinical trials to date have utilized an LV with an RV promoter and/or enhancer within the LTR.”
MethodInfluentialMontini, 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
Sessa, M., Lorioli, L., Fumagalli, F., Acquati, S., Redaelli, D., Baldoli, C., Canale, S., Lopez, I. D., Morena, F., Calabria, A., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30374-9 · Cited by 502
Maternal imprinting at the H19-Igf2 locus maintains adult haematopoietic stem cell quiescence
Venkatraman, A., He, X. C., Thorvaldsen, J. L., Sugimura, R., Perry, J. M., Tao, F., Zhao, M., Christenson, M. K., Sanchez, R., Yu, J. Y., et al. (2013) · Nature · doi:10.1038/nature12303 · Cited by 296
Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors
Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., & Baum, C. (2008) · Molecular Therapy · doi:10.1038/mt.2008.5 · Cited by 271
Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.3 · Cited by 189
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Cited by
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Duncan, C. N., Bledsoe, J. R., Grzywacz, B., Beckman, A., Bonner, M., Eichler, F. S., Kühl, J.-S., Harris, M. H., Slauson, S., Colvin, R. A., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2405541 · Cited by 164
Metadata
- Authors
- Espinoza, D. A., Fan, X., Yang, D., Cordes, S. F., Truitt, L. L., Calvo, K. R., Yabe, I. M., Demirci, S., Hope, K. J., Hong, S. G., Krouse, A., Metzger, M., Bonifacino, A., Lu, R., Uchida, N., Tisdale, J. F., Wu, X., DeRavin, S. S., Malech, H. L., Donahue, R. E., Wu, C., & Dunbar, C. E.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2019-06
- Volume
- 27
- Issue
- 6
- Pages
- 1074-1086
- ISSN
- 1525-0016
- Cited by
- 46
- References
- 14
- Language
- en
- Licence
- Funders
- NHLBI Division of Intramural Research
- Scientific Research Training Program for Young Talents
- Union Hospital
- Tongji Medical College
- Huazhong University of Science and Technology, China
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