Ontology highlight
ABSTRACT:
SUBMITTER: Rabai A
PROVIDER: S-EPMC6439232 | biostudies-literature | 2019 Jun
REPOSITORIES: biostudies-literature
Rabai Aymen A Reisser Léa L Reina-San-Martin Bernardo B Mamchaoui Kamel K Cowling Belinda S BS Nicot Anne-Sophie AS Laporte Jocelyn J
Molecular therapy. Nucleic acids 20190227
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 or correction of a heterozygous mutation in the Dynamin 2 (DNM2) gene that causes the autosomal dominant form of centronuclear myopathies (CNMs), a rare muscle disorder belonging to the large group of ...[more]