Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52

Duchenne muscular dystrophy (DMD) is a fatal X-linked disease caused by mutations in the DMD gene, leading to complete absence of dystrophin and progressive degeneration of skeletal musculature and myocardium. In DMD patients and in a corresponding pig model with a deletion of DMD exon 52 (DMDΔ52),...

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Detalles Bibliográficos
Autores principales: Stirm, Michael, Shashikadze, Bachuki, Blutke, Andreas, Kemter, Elisabeth, Lange, Andreas, Stöckl, Jan B., Jaudas, Florian, Laane, Laeticia, Kurome, Mayuko, Keßler, Barbara, Zakhartchenko, Valeri, Bähr, Andrea, Klymiuk, Nikolai, Nagashima, Hiroshi, Walter, Maggie C., Wurst, Wolfgang, Kupatt, Christian, Fröhlich, Thomas, Wolf, Eckhard
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2023
Materias:
Biological Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10629550/
https://www.ncbi.nlm.nih.gov/pubmed/37428903
http://dx.doi.org/10.1073/pnas.2301250120

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10629550/
https://www.ncbi.nlm.nih.gov/pubmed/37428903
http://dx.doi.org/10.1073/pnas.2301250120

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