pA regulator is an RNA-based, non-immunogenic regulation system of gene expression that achieves up to 900-fold induction by using an inducer drug within the clinically safe dose range.
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References
Clackson, T. Regulated gene expression systems. Gene Ther. 7, 120–125 (2000). This review pinpoints the limitations in existing gene therapies and prerequisites to implement drug-regulated gene expression systems in future gene therapy development.
Yokobayashi, Y. Aptamer-based and aptazyme-based riboswitches in mammalian cells. Curr. Opin. Chem. Biol. 52, 72–78 (2019). This review highlights developments in synthetic mammalian riboswitches that harness diverse regulatory mechanisms.
Tickner, Z. J. & Farzan, M. Riboswitches for controlled expression of therapeutic transgenes delivered by adeno-associated viral vectors. Pharmaceuticals 14, 554 (2021). This review discusses how adeno-associated viral vector (AAV)-delivered therapeutic systems might benefit from riboswitch regulation.
Berens, C., Thain, A. & Schroeder, R. A tetracycline-binding RNA aptamer. Bioorg. Med. Chem 9, 2549–2556 (2001). This paper describes the selection and isolation of the synthetic tetracycline-binding aptamer.
Strobel, B. et al. A small-molecule-responsive riboswitch enables conditional induction of viral vector-mediated gene expression in mice. ACS Synth Biol 9, 1292–1305 (2020). This paper evaluates an AAV-delivered tetracycline-responding riboswitch in mice, with pharmacokinetic studies in different tissues.
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This is a summary of: Luo, L., Jea, J. D.-Y., Wang, Y., Chao, P.-W. & Yen, L. Control of mammalian gene expression by modulation of polyA signal cleavage at 5′ UTR. Nat. Biotechnol. https://doi.org/10.1038/s41587-023-01989-0 (2024).
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A step closer to safe and efficient mammalian gene regulation. Nat Biotechnol (2024). https://doi.org/10.1038/s41587-023-02020-2
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DOI: https://doi.org/10.1038/s41587-023-02020-2
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