Suppressor tRNAs restore full-length dystrophin in mice with Duchenne muscular dystrophy

The latest chemistry news, including important research advances, business and policy trends, chemical safety practices, career guidance, and more.

There is no cure for Duchenne muscular dystrophy (DMD), in which affected individuals have muscle weakness and heart and respiratory issues. But for some, a new kind of gene therapy is showing promising early signs.

Roughly 15% of DMD cases are due to a nonsense mutation, in which a codon for an amino acid is mutated into a stop codon, leading to prematurely terminated translation of the protein dystrophin. Researchers at Tevard Biosciences have developed a suppressor transfer RNA (tRNA) that can read through those nonsense mutations and produce full-length dystrophin proteins in mice ( Sci. Adv. 2026, DOI: 10.1126/sciadv.aeg3466).