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Exaluren Induces COL4A5 Readthrough Across 49 Nonsense Variants in Alport Syndrome

June 1, 2022

Omachi et al. published in iScience a systematic screen of exaluren across 49 COL4A5 nonsense variants, demonstrating readthrough activity across all variants tested, with some mutations showing greater than 40-fold enhancement. These data underpin the scientific rationale for the NMAS program.

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