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A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder caused by mutations in the DMD gene and the subsequent lack of dystrophin protein. Recently, phosphorodiamidate morpholino oligomer (PMO)-antisense oligonucleotides (ASOs) targeting exon 51 or 53 to reestablish the DMD reading...

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Detalles Bibliográficos
Autores principales: Echigoya, Yusuke, Trieu, Nhu, Duddy, William, Moulton, Hong M., Yin, HaiFang, Partridge, Terence A., Hoffman, Eric P., Kornegay, Joe N., Rohret, Frank A., Rogers, Christopher S., Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8657897/
https://www.ncbi.nlm.nih.gov/pubmed/34884867
http://dx.doi.org/10.3390/ijms222313065