Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs.

There was a relative increase in PAX6 in ataluren-treated samples, but it did not reach statistical significance (0.90 ± 0.50, p = 0.22) ( Figure 3 B).

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12 , 13 A phase I/II clinical trial (ClinicalTrials.gov: NCT02647359 ) for aniridia was completed but failed to meet the primary endpoint despite showing a positive trend towards functional improvement ( https://www.prnewswire.com/news-releases/ptc-therapeutics-reports-fourth-quarter-and-full-year-2019-financial-results-and-provides-a-corporate-update-301014669.html ).

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AN2 iPSC-LESCs showed similar trends of increased PAX6 protein when dosed with TRIDs but failed to reach significance when treated with ataluren or amlexanox ( Figure 5 A).

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