Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide
N. Watanabe, Y. Tone, T. Nagata, S. Masuda, T. Saito, N. Motohashi, K. Takagaki, Y. Aoki, 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.
bioRxiv · Feb 23, 2023 · PreprintCell Biology