Delivery of A Chemically Modified Noncoding RNA Domain Improves Dystrophic Myotube Function (Adv. Sci. 20/2025)

Muscle RNA CYTOR is a pro‐myogenic long noncoding RNA in skeletal muscle. Chemical probing of CYTOR identifies exon 2 as an independent myogenic domain that promotes muscle function. Chemical engineering of the myogenic exon 2 RNA improves pharmacologic properties (RNA stability, immunogenicity) and delivery of the chemically‐enhanced, synthetic RNA domain improves disease characteristics in myotubes derived from Duchenne muscular dystrophy patients. More details can be found in article number 2410908 by Martin Wohlwend and co‐workers.