Bridging Duchenne muscular dystrophy therapy from rhesus monkeys to patients

Description

Guo and colleagues described their program to carry Duchenne muscular dystrophy therapies from rhesus monkeys toward patients, in a 2026 perspective. The disease, a progressive muscle wasting caused by loss of the protein dystrophin, involves thousands of variants and effects of age and disease stage. Mouse results alone cannot predict patient benefit. The team built monkey models by editing the dystrophin gene with CRISPR.

Guo recounts that their engineered monkeys carry a mutation in the common exon 45 to 55 hotspot and can be produced in under a year instead of six or seven. The monkeys develop degeneration, fibrosis, and motor impairment resembling early human disease, and revealed mechanisms including early immune activation and stem-cell dysfunction. On this platform the group tested circular RNA strategies that force exon skipping to restore the reading frame. One strategy is a single-cut DNA editing approach using a compact enzyme, bridging toward clinical application.

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Source: https://europepmc.org/article/MED/PMC13499363