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2018•NeurologyRequires access

Development of PPMO for the Treatment of Duchenne Muscular Dystrophy (P5.430)

Marco A. Passini, Li Gan, Jenna A. Wood, Monica Yao, Nelsa L. Estrella, Chris M. Treleaven, Bruce M. Wentworth, Jay S. Charleston, Joseph V. Rutkowski, Gunnar J. Hanson

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Abstract

To evaluate exon skipping and dystrophin production in muscle from mdx mice and cynomolgus monkeys following injection of cell-penetrating peptides (CPPs) conjugated to phosphorodiamidate morpholino oligomers (PPMOs) or unconjugated PMOs.

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What this paper is about

To evaluate exon skipping and dystrophin production in muscle from mdx mice and cynomolgus monkeys following injection of cell-penetrating peptides (CPPs) conjugated to phosphorodiamidate morpholino oligomers (PPMOs) or unconjugated PMOs.

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Available abstract

To evaluate exon skipping and dystrophin production in muscle from mdx mice and cynomolgus monkeys following injection of cell-penetrating peptides (CPPs) conjugated to phosphorodiamidate morpholino oligomers (PPMOs) or unconjugated PMOs.

Key concepts: Duchenne muscular dystrophy, Muscular dystrophy, Medicine, Art, Gerontology, Gynecology, Internal medicine

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