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J. Rousseau

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Open access Aug 2026

Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts

An in vitro prime editing workflow is established to generate and subsequently correct the mdx5cv mutation in mouse C2C12 myoblasts and provides proof of concept that precise correction of the pathogenic mutation is associated with restoration of dystrophin expression following myogenic differentiation.

A. Siddika, Fatima El Husseiny, J. Rousseau et al. · 0 citations

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