Niche-reprogramming CAR T therapy improves skeletal muscle regeneration and function in preclinical murine models of Duchenne muscular dystrophy.

uPAR-directed CAR T cells selectively eliminate uPAR+ macrophage and fibro-adipogenic progenitor subsets in Duchenne muscular dystrophy, improving dystrophic muscle regeneration and force production in preclinical murine models.

• Why it matters: Dystrophic muscle function and regeneration are impaired by a pathological microenvironment driven by chronic inflammation and fibrosis, and existing dystrophin-restoring approaches do not address these niche problems.
• What they did: The study identified uPAR as a shared surface marker on pathogenic immune/progenitor subsets with pro-inflammatory and fibrogenic signatures, then tested uPAR-directed CAR T cells for selective depletion and microenvironment remodeling.
• The result: After treatment, dystrophic muscle regeneration and force production increased, and diaphragm and respiratory function improved, supporting uPAR CAR T cells as a niche-remodeling regenerative immunotherapy in mice.

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