A significant advance in DMD cell therapy

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British researchers have succeeded in significantly improving the grafting of myogenic progenitor cells (MPC) into the muscle fibre of mouse models of Duchenne muscular dystrophy:

  • they used a special gel (hydrogel) to stabilise the donor cells so that they would take better hold in the muscle of the recipient mice,
  • Stem cells from patients suffering from Duchenne muscular dystrophy (DMD) were corrected by genome editing (CRISPR) and transformed into MPC,
  • Injected into mdx nude mice, they were able to be revascularised and reconnected to neuromuscular junctions in a durable manner.

This highly encouraging work has revived interest in cell therapy for DMD. The authors note, however, that the results obtained vary from one muscle fibre segment to another.

 

Engineered human myogenic cells in hydrogels generate innervated vascularized myofibers within dystrophic mouse muscle on long-term engraftment. Kowala A, Boot J, Meng J et al. Cell Rep Med. 2025 Mars.

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