Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping is a therapeutic approach for DMD. It employs antisense oligonucleotides (AONs) to restore the disrupted open reading frame, allowing the pro...

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Vydáno v:Mol Ther
Hlavní autoři: Jirka, Silvana M.G., ’t Hoen, Peter A.C., Diaz Parillas, Valeriano, Tanganyika-de Winter, Christa L., Verheul, Ruurd C., Aguilera, Begona, de Visser, Peter C., Aartsma-Rus, Annemieke M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5763161/
https://ncbi.nlm.nih.gov/pubmed/29103911
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.10.004
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