Antisense oligonucleotides (ASOs) are promising for targeting diseases by modulating gene expression, even for genes previously considered "undruggable." ASOs enter cells through endocytosis but face challenges as they often get trapped in endosomes, limiting their therapeutic effect. Strategies to enhance endosomal escape, including using endosomal escape agents and non-viral delivery vehicles, are critical to increase their effectiveness. Despite some success, only 1-2% of ASOs reach the target mRNA in the …

more

This paper introduces a novel therapeutic approach for Duchenne Muscular Dystrophy (DMD) using NS-089/NCNP-02, an antisense oligonucleotide (ASO) designed to skip exon 44 of the dystrophin gene. DMD is a severe muscle disorder caused by mutations in the DMD gene, leading to a lack of dystrophin protein. Exon skipping helps to restore the reading frame of dystrophin, enabling the production of a shorter but functional dystrophin protein. The authors focused …

more