Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Exonic duplications account for 10%-15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct the most frequent (exon 2) duplication in the DMD ge...
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Elsevier,
2017-06-01T00:00:00Z.
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