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

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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Bibliographic Details
Main Authors: Corinne Betts, Amer F Saleh, Andrey A Arzumanov, Suzan M Hammond, Caroline Godfrey, Thibault Coursindel, Michael J Gait, Matthew JA Wood
Format: Article
Language:English
Published: Elsevier 2012-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
cardiac muscle
Duchenne muscular dystrophy
phosphorodiamidate morpholino oligomer
PMO internalizing peptide
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116300956

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