Article
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2025
van Tienen Florence H J, Hoeijmakers Janneke G J, van der Leij Christiaan, Timmer Erika, Wanders Nikki, Lindsey Patrick J, Yi Fangzheng, Lin Fong, Kortekaas Susanne P M, Roelofs Helene, Westra Inge M, Meij Pauline, Wijnen Lambert A C M, de Coo Irenaeus F M, Smeets Hubert J M
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...
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