The use of CAR-T cells to support gene therapy in DMD

Researchers from Généthon (Evry) used genetically modified T lymphocytes (FAP-type CAR-T) to improve gene-drug transfers via adeno-associated viruses (AAV) in gene therapy:

  • after two injections, these CAR-T cells were found to be capable of reducing the fibrotic phenomena observed in the mouse model of Duchenne muscular dystrophy (DMD),
  • they enable the depletion of progenitor cells with high fibro-adipogenic potential from the muscle,
  • when applied in gene transfer protocols in this same model, gene therapy was improved.

This work illustrates the growing interaction between cell therapy and gene therapy.

 

FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy. Ferrand M, Rocca CJ, Corre G et al. Mol Ther Methods Clin Dev. 2025 Jul