Enhanced splicing modulation by NMA-modified antisense oligonucleotides
Karen Ling, Thazha P. Prakash, Jinghua Yu, Michaela Jackson, Seung Joo Chun, Gemma Bachmann, Noah M. Post, Sarah Greenlee, Armand Soriano, John L. Hunyara, Daniel A Norris, Paymaan Jafar‐Nejad, Eric E. Swayze, C. Frank Bennett, Frank Rigo
This paper introduces NMA-modified antisense oligonucleotides that show improved potency compared to MOE-modified SSOs in modulating splicing for SMA and Dravet syndrome.
Nucleic Acids Research · May 2, 2026Peer-reviewedPharmacology & Toxicology