Rinsho Shinkeigaku (Clinical Neurology)

Symposium 14

Exon Skipping Approach to Duchenne Muscular Dystorphy

Shin'ichi Takeda, M.D., Ph.D.1)

1)Translational Medical Center, National Center of Neurology and Psychiatry

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.
Full Text of this Article in Japanese PDF (464K)

(CLINICA NEUROL, 54: 1071|1073, 2014)
key words: Duchenne muscular dystrophy, dystrophin, exon skipping, antisense-oligonucleotides

(Received: 23-May-14)