Ontology highlight
ABSTRACT:
SUBMITTER: Tsoumpra MK
PROVIDER: S-EPMC6642283 | biostudies-literature | 2019 Jul
REPOSITORIES: biostudies-literature
Tsoumpra Maria K MK Fukumoto Seiji S Matsumoto Toshio T Takeda Shin'ichi S Wood Matthew J A MJA Aoki Yoshitsugu Y
EBioMedicine 20190627
Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphorodiamidate morpholino backbone (PMO) of ASOs generated the peptide-conjugated PMOs (PPMOs) that exhibit a dramatically improved pharmacokinetic profile. When tested in animal models, PPMOs demonstrate ...[more]