Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy

Antisense oligonucleotides (AONs) used to reframe dystrophin mRNA transcripts for Duchenne muscular dystrophy (DMD) patients are tested in clinical trials. Here, AONs are administered subcutaneously and intravenously, while the less invasive oral route would be preferred. Oral delivery of encapsulat...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: van Putten, Maaike, Young, Courtney, van den Berg, Sjoerd, Pronk, Amanda, Hulsker, Margriet, Karnaoukh, Tatyana G, Vermue, Rick, van Dijk, Ko Willems, de Kimpe, Sjef, Aartsma-Rus, Annemieke
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4459548/
https://ncbi.nlm.nih.gov/pubmed/25405468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.62
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