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

• Main Authors: Aymen Rabai (Author), Léa Reisser (Author), Bernardo Reina- (Author), Kamel Mamchaoui (Author), Belinda S. Cowling (Author), Anne-Sophie Nicot (Author), Jocelyn Laporte (Author)
• Format: Book
• Published: Elsevier, 2019-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...