AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.
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Acknowledgements
This work was supported by the European Union–NextGenerationEU through the Italian Ministry of University and Research under PNRR-M4C2-I1.3 project PE_00000019 ‘HEAL ITALIA’ to G.D’U. CUP J33C22002920006 and by the Italian Ministry of Health (RC-2024-2790614). The views and opinions expressed are those of the authors only and do not necessarily reflect those of the European Union or the European Commission. Neither the European Union nor the European Commission can be responsible for them.
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G.D’U. is a co-inventor on a patent related to the delivery of agents that enhance the activity or the expression of ERBB2 to promote cardiac regeneration, but declares no competing financial interests.
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D’Uva, G. A tunable gene therapy for heart regeneration. Nat Cardiovasc Res 4, 794–796 (2025). https://doi.org/10.1038/s44161-025-00666-6
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DOI: https://doi.org/10.1038/s44161-025-00666-6
