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Gene therapy for severe combined immunodeficiency: are we there yet?
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“In this regard, retroviral integration in the proximity of proto-oncogenes, particularly the LIM domain only 2 (LMO2) promoter, has been shown to be involved in leukemogenesis in 3 of the French patients and one British patient[41,43,49].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [49]
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In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
Gene therapy restores vision in a canine model of childhood blindness
Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202
IMMUNOLOGICAL RECONSTITUTION OF SEX-LINKED LYMPHOPENIC IMMUNOLOGICAL DEFICIENCY
Gatti, R., Meuwissen, H., Allen, H., Hong, R., & Good, R. (1968) · The Lancet · doi:10.1016/s0140-6736(68)92673-1 · Cited by 1,018
Severe combined immunodeficiency caused by deficiency in either the δ or the ε subunit of CD3
Basile, G. D. S., Geissmann, F., Flori, E., Uring-Lambert, B., Soudais, C., Cavazzana-Calvo, M., Durandy, A., Jabado, N., Fischer, A., & Deist, F. L. (2004) · Journal of Clinical Investigation · doi:10.1172/jci200422588 · Cited by 171
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Metadata
- Authors
- Cavazzana-Calvo, M., & Fischer, A.
- Venue
- Journal of Clinical Investigation
- Published
- 2007-06-01
- Volume
- 117
- Issue
- 6
- Pages
- 1456-1465
- ISSN
- 0021-9738
- Cited by
- 183
- References
- 5
- Language
- en
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