(2025)Cite this article
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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Booth, B. J. et al. RNA editing: expanding the potential of RNA therapeutics. Mol. Ther. 31, 1533–1549 (2023). A review article that summarizes the emerging field of therapeutic RNA editing and discusses key challenges in clinical development.
Teixeira, A. P. & Fussenegger, M. Synthetic gene circuits for regulation of next-generation cell-based therapeutics. Adv. Sci. 11, 1–24 (2024). A review article that presents examples of engineered genetic circuits applied to cell-based therapeutics.
Song, J., Zhuang, Y. & Yi, C. Programmable RNA base editing via targeted modifications. Nat. Chem. Biol. 20, 277–290 (2024). A review article that summarizes some emerging RNA editors and discusses their limitations, opportunities and future directions.
Yu, J. et al. Programmable RNA base editing with photoactivatable CRISPR-Cas13. Nat. Commun. 15, 1–14 (2024). This paper reports a light-inducible RNA base editor that employs a photoactivatable split dCas13b in conjugation with intact ADAR2dd variant.
Stroppel, A. S., Lappalainen, R. & Stafforst, T. Controlling site-directed RNA editing by chemically induced dimerization. Chemistry 27, 12300–12304 (2021). This paper reports a chemically inducible RNA base editor named the gibberellic acid (GA3)-induced SNAP-ADAR system.
Anadón, C. et al. Gene amplification-associated overexpression of the RNA editing enzyme ADAR1 enhances human lung tumorigenesis. Oncogene 35, 4407–4413 (2016). This paper reports the carcinogenic risk of overexpressing ADAR proteins.
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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).
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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DOI: https://doi.org/10.1038/s41587-025-02621-z