Genome editing for Duchenne muscular dystrophy: a glimpse of the future?

Mutations in Dystrophin, one of the largest proteins in the mammalian body, are causative for a severe form of muscle disease, Duchenne Muscular Dystrophy (DMD), affecting not only skeletal muscle, but also the heart. In particular, exons 45–52 constitute a hotspot for DMD mutations. A variety of mo...

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Detalles Bibliográficos
Autores principales: Kupatt, Christian, Windisch, Alina, Moretti, Alessandra, Wolf, Eckhard, Wurst, Wolfgang, Walter, Maggie C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455335/
https://www.ncbi.nlm.nih.gov/pubmed/33531685
http://dx.doi.org/10.1038/s41434-021-00222-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8455335/
https://www.ncbi.nlm.nih.gov/pubmed/33531685
http://dx.doi.org/10.1038/s41434-021-00222-4

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