Prednisolone Treatment Does Not Interfere with 2′-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy

In Duchenne muscular dystrophy (DMD), dystrophin deficiency leading to progressive muscular degeneration is caused by frame-shifting mutations in the DMD gene. Antisense oligonucleotides (AONs) aim to restore the reading frame by skipping of a specific exon(s), thereby allowing the production of a s...

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Autores principales: Verhaart, Ingrid E.C., Heemskerk, Hans, Karnaoukh, Tatyana G., Kolfschoten, Ingrid G.M., Vroon, Anne, van Ommen, Gert-Jan B., van Deutekom, Judith C.T., Aartsma-Rus, Annemieke
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2012
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Research Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3300076/
https://ncbi.nlm.nih.gov/pubmed/22017442
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.127
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