Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

• Main Authors: Shouta Miyatake (Author), Yoshitaka Mizobe (Author), Maria K. Tsoumpra (Author), Kenji Rowel Q. Lim (Author), Yuko Hara (Author), Fazel Shabanpoor (Author), Toshifumi Yokota (Author), Shin'ichi Takeda (Author), Yoshitsugu Aoki (Author)
• Format: Book
• Published: Elsevier, 2019-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 be...