Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes

Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more...

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Detalles Bibliográficos
Autores principales: Pires, Vanessa Borges, Simões, Ricardo, Mamchaoui, Kamel, Carvalho, Célia, Carmo-Fonseca, Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5524367/
https://www.ncbi.nlm.nih.gov/pubmed/28742140
http://dx.doi.org/10.1371/journal.pone.0181065

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