Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient.

ABSTRACT:

Background

Antisense oligonucleotide-induced exon skipping is a promising approach for treatment of Duchenne muscular dystrophy (DMD). We have systemically administered an antisense phosphorodiamidate morpholino oligomer (PMO) targeting dystrophin exons 6 and 8 to a dog with canine X-linked muscular dystrophy in Japan (CXMD(J)) lacking exon 7 and achieved recovery of dystrophin in skeletal muscle. To date, however, antisense chemical compounds used in DMD animal models have not been directly applied to a DMD patient having the same type of exon deletion. We recently identified a DMD patient with an exon 7 deletion and tried direct translation of the antisense PMO used in dog models to the DMD patient's cells.

Methodology/principal findings

We converted fibroblasts of CXMD(J)

SUBMITTER: Saito T 

PROVIDER: S-EPMC2923599 | biostudies-literature | 2010 Aug

REPOSITORIES: biostudies-literature