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Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells
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“A novel LVV was developed for the treatment of CGD by investigators in the UK, Germany, and Spain, using the promoter elements for two genes expressed in myeloid cells (Cathepsin G and cfes) to drive the CYBB cDNA, to avoid trans-activation of genes adjacent to integration sites in HSCs, but express the normal transgene in the maturing myeloid cells.39”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [39]
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T cells express a phagocyte-type NADPH oxidase that is activated after T cell receptor stimulation
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Cloning of a 67-kD Neutrophil Oxidase Factor with Similarity to a Noncatalytic Region of p60 c-src
Leto, T. L., Lomax, K. J., Volpp, B. D., Nunoi, H., Sechler, J. M. G., Nauseef, W. M., Clark, R. A., Gallin, J. I., & Malech, H. L. (1990) · Science · doi:10.1126/science.1692159 · Cited by 386
Self-citationHematopoietic lineage- and stage-restricted expression of the ETS oncogene family member PU.1
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Cited by
De Ravin, S. S., Li, L., Wu, X., Choi, U., Allen, C., Koontz, S., Lee, J., Theobald-Whiting, N., Chu, J., Garofalo, M., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aah3480 · Cited by 258
Self-citationGenetic disorders coupled to ROS deficiency
O’Neill, S., Brault, J., Stasia, M.-J., & Knaus, U. G. (2015) · Redox Biology · doi:10.1016/j.redox.2015.07.009 · Cited by 164
Retrovirus and lentivirus vector design and methods of cell conditioning
Cooray, S., Howe, S. J., & Thrasher, A. J. (2012) · Methods in Enzymology · doi:10.1016/b978-0-12-386509-0.00003-x · Cited by 65
Self-citationEx 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
- Santilli, G., Almarza, E., Brendel, C., Choi, U., Beilin, C., Blundell, M. P., Haria, S., Parsley, K. L., Kinnon, C., Malech, H. L., Bueren, J. A., Grez, M., & Thrasher, A. J.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2011-01
- Volume
- 19
- Issue
- 1
- Pages
- 122-132
- ISSN
- 1525-0016
- Cited by
- 144
- References
- 8
- Language
- en
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