Elimination of Mutant mtDNA by an Optimized mpTALEN Restores Differentiation Capacities of Heteroplasmic MELAS-iPSCs
Naoki Yahata, Hiroko Boda, Ryuji Hata
Abstract
, which was relieved by decreasing the heteroplasmy level with G13513A-mpTALEN. Additionally, drug-inducible, myogenic differentiation 1 (MYOD)-transfected MELAS-iPSCs (MyoD-iPSCs) efficiently differentiated into myosin heavy chain-positive myocytes, with or without mutant mtDNA. Hence, heteroplasmic MyoD-iPSCs controlled by fine-tuned mpTALENs may contribute to a detailed analysis of the relationship between mutation load and cellular phenotypes in disease modeling.
Topics & Concepts
HeteroplasmyMitochondrial DNABiologyMELAS syndromeInduced pluripotent stem cellMitochondrial myopathyLactic acidosisGeneticsMolecular biologyMitochondrial diseaseMyoDCell biologyCellular differentiationBiochemistryGeneEmbryonic stem cellMitochondrial Function and PathologyCRISPR and Genetic EngineeringRNA and protein synthesis mechanisms