Ontology highlight
ABSTRACT:
SUBMITTER: Miyatake S
PROVIDER: S-EPMC6374502 | biostudies-literature | 2019 Mar
REPOSITORIES: biostudies-literature
Miyatake Shouta S Mizobe Yoshitaka Y Tsoumpra Maria K MK Lim Kenji Rowel Q KRQ Hara Yuko Y Shabanpoor Fazel F Yokota Toshifumi T Takeda Shin'ichi S Aoki Yoshitsugu Y
Molecular therapy. Nucleic acids 20190125
Exon skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular dystrophy (DMD). The most significant limitation of these clinically used compounds is their lack of delivery systems that target muscles; thus, cell-penetrating peptides are being developed to enhance uptake into muscles. Recently, we reported that uptake of peptide-conjugated PMOs into myofibers was mediated by scavenger receptor class A (SR-A), which binds negatively char ...[more]