van Putten, Maaike <br/><br/>
Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.  [electronic resource] 

 -  Nucleic acid therapeutics  04 2019 
 - 92-103 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>2159-3345 
<br/><br/><label>Standard No.: </label>10.1089/nat.2018.0759  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Animals<br/>Clinical Trials as Topic<br/>Dystrophin--antagonists & inhibitors<br/>Exons--drug effects<br/>Genetic Therapy<br/>Humans<br/>Mice<br/>Mice, Inbred mdx<br/>Morpholinos--genetics<br/>Muscular Atrophy, Spinal<br/>Muscular Dystrophy, Duchenne--genetics<br/>Oligonucleotides--pharmacology<br/>Oligonucleotides, Antisense--genetics<br/>Organothiophosphorus Compounds--pharmacology<br/>Utrophin--genetics