Article

Pluripotent stem cell-based drug discovery uncovers sildenafil as a treatment for mitochondrial disease

2025-05-16

Abstract excerpt

Mitochondrial disease encompasses inherited disorders affecting mitochondrial function. A severe and untreatable form of mitochondrial disease is Leigh syndrome (LS) causing psychomotor regression and metabolic crises. To accelerate drug discovery for LS, we screened a library of 5,632 repurposable compounds in induced pluripotent stem cell (iPSC)-derived neural progenitor cells (NPCs) from LS patients. We identif...

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Literature Corpus work: 61f76b20-ad50-51f5-aaa3-980f5d3804ed
DOI: 10.1101/2025.05.15.25325571

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