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Activating internal ribosome entry to treat Duchenne muscular dystrophy

Mutations in the DMD gene, encoding dystrophin, cause the most common forms of muscular dystrophy. A new study shows that forcing translation of DMD from an internal ribosome entry site can alleviate Duchenne muscular dystrophy symptoms in a mouse model.

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Figure 1: Exon 2 skipping in a DMD mouse model with an exon 2 duplication can ameliorate the DMD phenotype.

Debbie Maizels/Nature Publishing Group


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Correspondence to Kathryn N North.

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The authors declare no competing financial interests.

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Lamandé, S., North, K. Activating internal ribosome entry to treat Duchenne muscular dystrophy. Nat Med 20, 987–988 (2014).

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