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Tunable gene control via RNA splicing with a clinically approved small molecule

et al. developed a DNA sequence module which enables splicing-mediated control of gene expression via an inducer which is already an FDA-approved medicine. An extremely useful advance for gene therapy since it allows activation of therapeutic genes only when we want them to turn on. I expect this kind of tool will be used heavily by the biomedical industry in coming years.


Precise transgene regulation is crucial for the safety and efficacy of next-generation therapies. Here, authors develop RisdiON, a compact gene switch controlled by the approved oral drug risdiplam, showing this system allows for tunable, reversible regulation across diverse applications in vitro and in vivo.

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