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

Molecular Therapy - Nucleic Acids

Volumn 7, Issue , 2017, Pages 11-19

  Lattanzi, Annalisa ^a   Moiani, Arianna ^a   Izmiryan, Araksya ^a   Martin, Samia ^a   Mavilio, Fulvio ^a   Bovolenta, Matteo ^a,c   Duguez, Stephanie ^b   Mamchaoui, Kamel ^b   Mouly, Vincent ^b   Barbon, Elena ^c   Bernardi, Francesco ^c  

^a GÉNÉTHON   (France)

^b Sorbonne Université   (France)

^c UNIVERSITY OF FERRARA   (Italy)

Author keywords

CRISPR Cas9; duplication; dystrophin; gene editing; lentivirus

Indexed keywords

DYSTROPHIN; GUIDE RNA;

ARTICLE; CONTROLLED STUDY; CRISPR CAS SYSTEM; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE DELETION; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; INTRON; MYOBLAST; MYOTUBE; PRIORITY JOURNAL; PROTEIN EXPRESSION;

EID: 85020698228     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1016/j.omtn.2017.02.004     Document Type: Article

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