Article

High-throughput discovery of arginine-depleted peptides enables effective antisense delivery for Duchenne muscular dystrophy

2026-06-11

Abstract excerpt

Phosphorodiamidate morpholino oligomers (PMOs) are approved exon-skipping antisense therapeutics for Duchenne muscular dystrophy (DMD), but their clinical utility is limited by poor uptake in muscle tissue, necessitating frequent high-dose administration. Cell-penetrating peptides (CPPs) can enhance intracellular delivery of PMOs, yet conventional arginine-rich CPPs often cause dose-limiting toxicity, including re...

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Literature Corpus work: 5c9030a6-08c6-539f-b929-4d0137005997
DOI: 10.64898/2026.06.07.730741

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