Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse.

ABSTRACT: The severe muscle wasting disorder Duchenne muscular dystrophy (DMD) is characterized by the absence of dystrophin, a protein that is essential for muscle stability. Restoring this protein has therapeutic potential. Antisense oligonucleotides (ASOs), designed to target and skip exons, can restore the reading frame that is disrupted in these patients, enabling the production of partially functional dystrophin. Achieving optimal dystrophin restoration remains challenging due to limited delivery and cellular uptake. Muscle homing peptides conjugated to ASOs are a way to achieve this. Previously, CyPep10 (CP10) has been used to significantly increase exon skipping efficiency for the 2'-O-methyl phosphorothioate chemistry in the mdx mouse model for DMD. Here, we explore the effect of usi

SUBMITTER: Schneider AE 

PROVIDER: S-EPMC12329520 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature