science π¬ New DNA tech shows promise for DMD treatment
A supercharged oligonucleotide therapeutic has shown potential for Duchenne muscular dystrophy (DMD) in preclinical studies. Researchers from Nanjing Antisense Biopharm used bifunctional antisense oligonucleotides (ASOs) in mouse models of the disease. The 5D-ASO technique, featuring an 8-nt tail, robustly enhanced exon skipping in skeletal and cardiac muscles. This approach restored dystrophin expression and showed a favorable safety profile in cynomolgus monkeys. These results position bipartite-ASO technology as a powerful platform for exon-skipping therapeutics.