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

• Main Authors: Gang Han (Author), Ben Gu (Author), Caorui Lin (Author), Hanhan Ning (Author), Jun Song (Author), Xianjun Gao (Author), Hong M. Moulton (Author), HaiFang Yin (Author)
• Format: Book
• Published: Elsevier, 2019-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...