Concepedia

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

Hirofumi Komaki, Takashi Saito, S. Masuda, Eri Takeshita, Masayuki Sasaki, Hisateru Tachimori, Harumasa Nakamura, Yoshitsugu Aoki, Shin’ichi Takeda

Science Translational Medicine · 2018 · 153 citations · 23 references

DOI

Concepts

Abstract

NS-065/NCNP-01, an antisense oligonucleotide that enables exon 53 skipping in the dystrophin gene, showed a favorable safety profile and promising pharmacokinetics in 10 patients with Duchenne muscular dystrophy.

References

23

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Eric P. Hoffman, Robert H. Brown, Louis M. Kunkel · Cell · 1987 · 4.7K citations

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Çirak, Virginia Arechavala‐Gomeza, Michela Guglieri et al. · The Lancet · 2011 · 862 citations · Full text

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Evidence‐based path to newborn screening for duchenne muscular dystrophy

Jerry R. Mendell, Chris Shilling, Nancy D. Leslie et al. · Annals of Neurology · 2012 · 817 citations

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Eteplirsen for the treatment of Duchenne muscular dystrophy

Jerry R. Mendell, Louise R. Rodino‐Klapac, Zarife Sahenk et al. · Annals of Neurology · 2013 · 795 citations · Full text

, , +14

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Judith C. van Deutekom, Anneke A. M. Janson, Ieke B. Ginjaar et al. · New England Journal of Medicine · 2007 · 762 citations

, , +15