Rebs Sabine, Streckfuss-Bömeke Katrin
Institute of Pharmacology and Toxicology, University of Würzburg, Würzburg, Germany.
Clinic for Cardiology and Pneumology, University Medicine Göttingen and DZHK (German Centre for Cardiovascular Research), Göttingen, Germany.
Front Mol Med. 2023 Sep 1;3:1222986. doi: 10.3389/fmmed.2023.1222986. eCollection 2023.
Mutations in the mitochondrial-DNA or mitochondria related nuclear-encoded-DNA lead to various multisystemic disorders collectively termed mitochondrial diseases. One in three cases of mitochondrial disease affects the heart muscle, which is called mitochondrial cardiomyopathy (MCM) and is associated with hypertrophic, dilated, and noncompact cardiomyopathy. The heart is an organ with high energy demand, and mitochondria occupy 30%-40% of its cardiomyocyte-cell volume. Mitochondrial dysfunction leads to energy depletion and has detrimental effects on cardiac performance. However, disease development and progression in the context of mitochondrial and nuclear DNA mutations, remains incompletely understood. The system of induced pluripotent stem cell (iPSC)-derived cardiomyocytes (CM) is an excellent platform to study MCM since the unique genetic identity to their donors enables a robust recapitulation of the predicted phenotypes in a dish on a patient-specific level. Here, we focus on recent insights into MCM studied by patient-specific iPSC-CM and further discuss research gaps and advances in metabolic maturation of iPSC-CM, which is crucial for the study of mitochondrial dysfunction and to develop novel therapeutic strategies.
线粒体DNA或与线粒体相关的核编码DNA的突变会导致各种多系统疾病,统称为线粒体疾病。三分之一的线粒体疾病病例会影响心肌,这被称为线粒体心肌病(MCM),并与肥厚型、扩张型和致密化不全型心肌病相关。心脏是一个能量需求很高的器官,线粒体占其心肌细胞体积的30%-40%。线粒体功能障碍会导致能量消耗,并对心脏功能产生有害影响。然而,线粒体和核DNA突变背景下的疾病发展和进展仍未完全了解。诱导多能干细胞(iPSC)衍生的心肌细胞(CM)系统是研究MCM的一个优秀平台,因为它们与供体独特的基因身份能够在培养皿中以患者特异性水平有力地重现预测的表型。在这里,我们重点关注通过患者特异性iPSC-CM对MCM的最新见解,并进一步讨论iPSC-CM代谢成熟方面的研究差距和进展,这对于研究线粒体功能障碍和开发新的治疗策略至关重要。