title: [Exon skipping approach to Duchenne muscular dystorphy].
abstract: Exon skipping therapy by antisense oligonucleotide is a promising approach to Duchenne muscular dystrophy (DMD). We have reported the proof-of-concept studies using morpholino on mice or dog DMD model and on patient derived cells. Based on these results, we had promoted collaborative research with a Japanese pharmaceutical company and encouraged development of DMD gene exon 53 skipping drug, then finally started an investigator-initiated clinical trial from 2013. Furthermore, we are addressing exploratory researches to expand the possibility of AON; such as, an application of AON to Fukuyama congenital muscular dystrophy, and an elucidation of AON uptake mechanism.
authors: Translational Medical Center, National Center of Neurology and Psychiatry.
authors: Shin'ichi
authors: S
authors: Takeda
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D004285:Dogs; D005091:Exons / Q000235:genetics*; D006801:Humans; D051379:Mice; D058990:Molecular Targeted Therapy / Q000379:methods / Q000639:trends; D060172:Morpholinos / Q000627:therapeutic use; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*
chemical_list: D060172:Morpholinos; D016376:Oligonucleotides, Antisense