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...

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Detalles Bibliográficos
Autores principales: Rabai, Aymen, Reisser, Léa, Reina-San-Martin, Bernardo, Mamchaoui, Kamel, Cowling, Belinda S., Nicot, Anne-Sophie, Laporte, Jocelyn
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6439232/
https://www.ncbi.nlm.nih.gov/pubmed/30925452
http://dx.doi.org/10.1016/j.omtn.2019.02.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6439232/
https://www.ncbi.nlm.nih.gov/pubmed/30925452
http://dx.doi.org/10.1016/j.omtn.2019.02.019

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