Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results. [electronic resource]
Producer: 20200722Description: 92-103 p. digitalISSN:- 2159-3345
- Animals
- Clinical Trials as Topic
- Dystrophin -- antagonists & inhibitors
- Exons -- drug effects
- Genetic Therapy
- Humans
- Mice
- Mice, Inbred mdx
- Morpholinos -- genetics
- Muscular Atrophy, Spinal
- Muscular Dystrophy, Duchenne -- genetics
- Oligonucleotides -- pharmacology
- Oligonucleotides, Antisense -- genetics
- Organothiophosphorus Compounds -- pharmacology
- Utrophin -- genetics
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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