Dystrophin Gene Exon Deletion Using Engineered Nucleases
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Mar 12, 2015
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Mar 12, 2014
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The invention relates to the field of molecular biology and recombinant nucleic acid technology. In particular, the invention relates to methods of treating patients with Duchenne Muscular Dystrophy comprising the removal of at least one exon from the dystrophin gene using engineered nucleases to restore the normal reading frame. Further disclosed are engineered nucleases suitable for using the methods.
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