U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length <i>DMD</i> expression in patient-derived cell lines.

27 Translational development will require further studies, but this proof-of-concept study confirms that the pseudoexon class of DMD mutations is amenable to U7snRNA treatment and demonstrates the potential of such individualized gene therapies to restore full-length dystrophin expression, which may be significant and durable.

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