Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

Antisense oligonucleotides (AONs) are being developed as RNA therapeutic molecules for Duchenne muscular dystrophy. For oligonucleotides with the 2′-O-methyl-phosphorothioate (2OMePS) RNA chemistry, proof of concept has been obtained in patient-specific muscle cell cultures, the mouse and dog diseas...

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Autores principales: Heemskerk, Hans, de Winter, Christa, van Kuik, Petra, Heuvelmans, Niki, Sabatelli, Patrizia, Rimessi, Paola, Braghetta, Paola, van Ommen, Gert-Jan B, de Kimpe, Sjef, Ferlini, Alessandra, Aartsma-Rus, Annemieke, van Deutekom, Judith CT
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2010
Materias:
Original Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC2889733/
https://ncbi.nlm.nih.gov/pubmed/20407428
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.72
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