Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease typically caused by protein-truncating mutations that preclude synthesis of a functional dystrophin. Exonic deletions are the most common type of DMD lesion, however, whole exon duplications account for between 10–15% of all report...

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Detalles Bibliográficos
Autores principales: Greer, Kane, Johnsen, Russell, Nevo, Yoram, Fellig, Yakov, Fletcher, Susan, Wilton, Steve D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7350004/
https://www.ncbi.nlm.nih.gov/pubmed/32630425
http://dx.doi.org/10.3390/ijms21124511

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7350004/
https://www.ncbi.nlm.nih.gov/pubmed/32630425
http://dx.doi.org/10.3390/ijms21124511

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