Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

Duchenne muscular dystrophy is a severe, progressive muscle-wasting disease that is caused by mutations that abolish the production of functional dystrophin protein. The exon skipping approach aims to restore the disrupted dystrophin reading frame, to allow the production of partially functional dys...

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Detalles Bibliográficos
Autores principales: van Putten, Maaike, Tanganyika-de Winter, Christa, Bosgra, Sieto, Aartsma-Rus, Annemieke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2019
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6461150/
https://www.ncbi.nlm.nih.gov/pubmed/30672725
http://dx.doi.org/10.1089/nat.2018.0759

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6461150/
https://www.ncbi.nlm.nih.gov/pubmed/30672725
http://dx.doi.org/10.1089/nat.2018.0759

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