Exon Snipping in Duchenne Muscular Dystrophy

Dwi U Kemaladewi¹, Ronald D Cohn²

Affiliations

¹ Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
² Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada; Department of Pediatrics, University of Toronto, Toronto, ON, Canada; Division of Clinical and Metabolic Genetics, the Hospital for Sick Children, Toronto, ON, Canada; Centre for Genetic Medicine, the Hospital for Sick Children, Toronto, ON, Canada. Electronic address: ronald.cohn@sickkids.ca.

PMID: 26856237
DOI: 10.1016/j.molmed.2016.01.007

Comment

Exon Snipping in Duchenne Muscular Dystrophy

Dwi U Kemaladewi et al. Trends Mol Med. 2016 Mar.

. 2016 Mar;22(3):187-189.

doi: 10.1016/j.molmed.2016.01.007. Epub 2016 Feb 5.

Authors

Dwi U Kemaladewi¹, Ronald D Cohn²

Affiliations

¹ Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada.
² Genetics and Genome Biology Program, the Hospital for Sick Children, Toronto, ON, Canada; Department of Molecular Genetics, University of Toronto, Toronto, ON, Canada; Department of Pediatrics, University of Toronto, Toronto, ON, Canada; Division of Clinical and Metabolic Genetics, the Hospital for Sick Children, Toronto, ON, Canada; Centre for Genetic Medicine, the Hospital for Sick Children, Toronto, ON, Canada. Electronic address: ronald.cohn@sickkids.ca.

PMID: 26856237
DOI: 10.1016/j.molmed.2016.01.007

Abstract

Duchenne muscular dystrophy (DMD) is a life-limiting neuromuscular disorder caused by mutations in the DMD gene encoding dystrophin. We discuss very recent studies that used CRISPR/Cas9 technology to 'snip out' mutated exons in DMD, restoring the reading frame of the gene. We also present cautionary aspects of translating this exciting technology into clinical practice.

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA. Nelson CE, et al. Science. 2016 Jan 22;351(6271):403-7. doi: 10.1126/science.aad5143. Epub 2015 Dec 31. Science. 2016. PMID: 26721684 Free PMC article.

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Exon Snipping in Duchenne Muscular Dystrophy

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Exon Snipping in Duchenne Muscular Dystrophy

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Medical