A lack of broadly applicable methods to precisely control therapeutic gene expression is a key challenge for gene therapies. We present a technology that offers a photoactivatable RNA base editor for tunable and reversible regulation of gene expression, with implications for improving the safety and efficacy of gene therapy.
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Fig. 1: Optogenetic therapy for hemophilia B using PA-rABE.
References
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This is a summary of: Li, H. et al. Engineering a photoactivatable A-to-I RNA base editor for gene therapy in vivo. Nat. Biotechnol. https://doi.org/10.1038/s41587-025-02610-2 (2025).
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A light-inducible RNA base editor for precise gene expression. Nat Biotechnol (2025). https://doi.org/10.1038/s41587-025-02621-z
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Published:31 March 2025
DOI:https://doi.org/10.1038/s41587-025-02621-z
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