Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes

Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes

Genome editing with the CRISPR/Cas9 technology has emerged recently as a potential strategy for therapy in genetic diseases. For dominant mutations linked to gain-of-function effects, allele-specific correction may be the most suitable approach. In this study, we tested allele-specific inactivation...

Πλήρης περιγραφή

Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς: Aymen Rabai, Léa Reisser, Bernardo Reina-San-Martin, Kamel Mamchaoui, Belinda S. Cowling, Anne-Sophie Nicot, Jocelyn Laporte
Μορφή: Άρθρο
Γλώσσα:English
Έκδοση: Elsevier 2019-06-01
Σειρά:Molecular Therapy: Nucleic Acids
Διαθέσιμο Online:http://www.sciencedirect.com/science/article/pii/S2162253119300502

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