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

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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Bibliographic Details
Main Authors: Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
dystrophin
CRISPR/Cas9
duplication
lentivirus
gene editing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301300

Internet

http://www.sciencedirect.com/science/article/pii/S2162253117301300

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