Cell BiologybioRxivPreprint
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide
N. Watanabe, Y. Tone, T. Nagata…·S. Takeda
This study develops NS-089/NCNP-02, a dual-targeting ASO for exon 44 skipping in DMD, showing effective in vitro and in vivo exon skipping and dystrophin expression.
DOI 10.1101/2023.02.23.529798