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Hepatic gene replacement restores energy metabolism and doubles the survival in mouse model of GRACILE syndrome, a neonatal mitochondrial disease

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1. kallijar 08 May 2026
  
  in bioRxiv
  
  Now published in Molecular Therapy. https://www.sciencedirect.com/science/article/pii/S1525001626003096?via%3Dihub
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biorxiv.org/content/10.1101/2025.09.23.677965v1