A 'backup' protein, switched on with gene editing, steadied weakening muscles

In Duchenne muscular dystrophy, a missing protein called dystrophin leaves muscles fragile. The body keeps a near-copy in reserve — a protein called utrophin — but never makes enough of it to take over. A team at Penn Medicine and Children's Hospital of Philadelphia has now used gene editing to turn up utrophin production in dystrophic muscle, and the muscle worked better. The work, published in Molecular Therapy Advances, brought together the mRNA delivery expertise of Nobel laureate Drew Weissman and lipid nanoparticle specialist Michael Mitchell. It is early-stage research, but it points at a strategy that could help regardless of which dystrophin mutation a child carries.

SDG 3 · Good health and well-being — Mechanism: tech innovation