A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

Antisense RNA technology is a strategy for the treatment of Duchenne muscular dystrophy (DMD), a progressive and universally fatal X-linked neuromuscular disease caused by frameshift mutations in the gene encoding dystrophin. Phosphorodiamidate morpholino oligomers (PMOs) are an antisense RNA platfo...

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Bibliographic Details
Main Authors: Li Gan, Leslie C.L. Wu, Jenna A. Wood, Monica Yao, Chris M. Treleaven, Nelsa L. Estrella, Bruce M. Wentworth, Gunnar J. Hanson, Marco A. Passini
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: oligonucleotides: therapies and applications
Duchenne muscular dystrophy
dystrophin
exon-skipping therapy
neuromuscular disease
peptide-conjugated phosphorodiamidate morpholino oligomer
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122002189

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http://www.sciencedirect.com/science/article/pii/S2162253122002189

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