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Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide
This post introduces NS-089/NCNP-02, a novel exon-skipping therapy for Duchenne muscular dystrophy (DMD). Designed to target exon 44, this antisense oligonucleotide improves dystrophin restoration in DMD patients. Unlike traditional single-target ASOs, NS-089/NCNP-02 skips two different sequences within exon 44, enhancing efficiency. Studies in patient-derived cells and cynomolgus monkeys confirm its ability to induce exon skipping in both skeletal and cardiac muscle, offering a promising systemic therapy for DMD.
