Mutations that truncate the reading frame 5′ of exon 5 of the DMD gene result in use of an internal ribosome entry site (IRES). This element allows alternate translational initiation beginning within exon 6 that results in expression of an N-truncated isoform. Despite lacking half of the actin binding domain 1, this isoform is highly functional. We developed an AAV9.U7snRNA vector directed against exon 2 which induces skipping of this exon, thus resulting in a truncation of the reading frame therefore forcing expression of the highly functional N-truncated protein.

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