Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates.

Michael P. Carver,Jay S. Charleston,Courtney Shanks,Jianbo Zhang, Mark Mense,Alok K. Sharma,Harjeet Kaur,Peter Sazani

Journal of neuromuscular diseases（2016）

引用 16|浏览5

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摘要

Nonclinical results in NHPs for these three PMOs, together with the excellent clinical safety established for eteplirsen to date, suggest that once-weekly IV administration of PMOs for lifetime durations at therapeutic doses will be well tolerated by patients with DMD.

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关键词

AVI-4658,Duchenne muscular dystrophy,eteplirsen,exon skipping,kidney toxicity,non-human primates,phosphorodiamidate morpholino oligomers,splice switching oligonucleotides,toxicity studies

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