Correction of the Splicing Defect Caused by a Recurrent Variant in ABCA4 (c.769-784C>T) That Underlies Stargardt Disease

AON1, -4, and -6 targeting the cryptic SAS had the lowest splicing-correction efficacies and did not reach statistical significance ( p = 0.70, p = 0.29, and p = 0.30, respectively, when compared to AON-untreated, patient-derived cycloheximide control).

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The high success rate in PE exclusion, even with the AONs that failed to reach statistical significance, prompted us to re-test the same AONs in patient-derived fibroblasts. 3.2.

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