Repurposing VIPER to estimate differential splicing factor activity. This repository contains all the code to reproduce our analyses. Based on https://github.com ...
– 53.7% mean splicing correction observed following a single 15 mg/kg dose of PGN-EDODM1, with all patients showing an improvement in splicing – – PGN-EDODM1 was generally well-tolerated at 15 mg/kg, ...
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