762. Successful Use of Out-Of-Frame Exon 2 Skipping Induces IRES-Driven Expression of the N-Truncated Dystrophin Isoform: Promising Approach for Treating Other 5’ Dystrophin Mutations

Molecular Therapy - United States
doi 10.1016/s1525-0016(16)35775-6
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Elsevier BV

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