Article
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe – first phase I/II human clinical study
2025-01-18
Abstract excerpt
Progressive myopathy and exercise intolerance significantly impair quality of life in over 50% of m.3243A>G mutation carriers, with no curative therapy currently available. We hypothesize that intra-arterial administration of autologous, mtDNA mutation-free myogenic stem cells, mesoangioblasts, can reduce mutation load, enhance oxidative phosphorylation, and improve muscle function. To test this, the tibialis ante...
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Identifiers and source
- Literature Corpus work
- 370cb430-371e-591c-9157-17fa1f80bb2b
- DOI
- 10.1101/2025.01.17.24319434
