Translational readthrough therapy for ADPKD induces polycystin1 expression and partially rescues functional deficits in PKD1 mutant cells.

Sci Rep · 2025 · PMC12663426 · PMID 41274954

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highly significantno p-value reported

The efficacy of 400 μg/ml ELX-02 was slightly lower yet still highly significant: ~ 21% (clone A) and ~ 22% (clone B) relative to the wildtype Polycystin1 expression and ~ 40-fold (clone A) and ~ 50-fold (clone B) increase compared to the untreated HEK293 clones expressing PKD1-E3020X-FLAG cDNA.

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