Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

• Main Authors: Annalisa Lattanzi (Author), Stephanie Duguez (Author), Arianna Moiani (Author), Araksya Izmiryan (Author), Elena Barbon (Author), Samia Martin (Author), Kamel Mamchaoui (Author), Vincent Mouly (Author), Francesco Bernardi (Author), Fulvio Mavilio (Author), Matteo Bovolenta (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...