Hexose Potentiates Peptide-Conjugated Morpholino Oligomer Efficacy in Cardiac Muscles of Dystrophic Mice in an Age-Dependent Manner

Insufficient delivery of oligonucleotides to muscle and heart remains a barrier for clinical implementation of antisense oligonucleotide (AO)-mediated exon-skipping therapeutics in Duchenne muscular dystrophy (DMD), a lethal monogenic disorder caused by frame-disrupting mutations in the DMD gene. We...

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Bibliographic Details
Main Authors: Gang Han, Ben Gu, Caorui Lin, Hanhan Ning, Jun Song, Xianjun Gao, Hong M. Moulton, HaiFang Yin
Format: Article
Language:English
Published: Elsevier 2019-12-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119302598