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Activating internal ribosome entry to treat Duchenne muscular dystrophy

Nature Medicine volume 20, pages 987–988 (2014)Cite this article

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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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Authors and Affiliations

  1. Shireen R. Lamandé and Kathryn N. North are in the Murdoch Childrens Research Institute and Department of Paediatrics, University of Melbourne, Royal Children's Hospital, Melbourne, Victoria, Australia.,

    Shireen R Lamandé & Kathryn N North

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  1. Shireen R Lamandé
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  2. Kathryn N North
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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). https://doi.org/10.1038/nm.3677

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