Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. C...

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Autors principals: Betts, Corinne, Saleh, Amer F, Arzumanov, Andrey A, Hammond, Suzan M, Godfrey, Caroline, Coursindel, Thibault, Gait, Michael J, Wood, Matthew JA
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2012
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3438601/
https://ncbi.nlm.nih.gov/pubmed/23344180
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.30
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