Induced pluripotent stem cell-derived models for mtDNA diseases.

Abstract excerpt

Mitochondrial disease due to mutations in the mitochondrial DNA (mtDNA) is a common cause of human inherited disorders. Targeted modification of the mitochondrial genome has not succeeded with the current transgenic technologies. Furthermore, readily available cultured patient cells often do not manifest the disease phenotype. Therefore, pathogenic mechanisms underlying these disorders remain largely unknown, as...

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• Lipid Metabolism, Inborn Errors
• Acyl-CoA Dehydrogenase, Long-Chain
• Cell Culture Techniques
• Congenital Bone Marrow Failure Syndromes
• Induced Pluripotent Stem Cells