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Safe harbours for the integration of new DNA in the human genome
Abstract
Interactions between newly integrated DNA and the host genome limit the reliability and safety of transgene integration for therapeutic cell engineering and other applications. Although targeted gene delivery has made considerable progress, the question of where to insert foreign sequences in the human genome to maximize safety and efficacy has received little attention. In this Opinion article, we discuss 'genomic safe harbours' - chromosomal locations where therapeutic transgenes can integrate and function in a predictable manner without perturbing endogenous gene activity and promoting cancer.
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In humans, the region surrounding mROSA26 is also surrounded by proto-oncogenes that could influence transgene expression.
“In humans, this region is also surrounded by proto-oncogenes that could influence transgene expression.52”
From Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo (2024) · cites this paper as [52]
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A census of human cancer genes
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BackgroundTransfusion 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
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Metadata
- Authors
- Sadelain, M., Papapetrou, E. P., & Bushman, F. D.
- Venue
- Nature Reviews Cancer
- Published
- 2011-12-01
- Volume
- 12
- Issue
- 1
- Pages
- 51-58
- ISSN
- 1474-175X
- 1474-1768
- Cited by
- 441
- References
- 5
- Language
- en
- Licence
- www.springer.com/tdm (tdm)
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