Citation:
Rinsho Shinkeigaku. 2014;54(12):1071-3. doi: 10.5692/clinicalneurol.54.1071
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.
Epub:
Not Epub
Link to Publication:
http://www.neurology-jp.org/Journal/cgi-bin/journal.cgi?lg=eg&pg=ex&vl=54&no=12&tp=1071