Feil Family Brain and Mind Research Institute, Weill Cornell Medicine, 407 East 61st Street, New York, NY 10065, USA.
Neuroscience Graduate Program, Will Cornell Graduate School of Medical Sciences, 1300 York Ave, New York, NY 10065, USA.
Dis Model Mech. 2023 Oct 1;16(10). doi: 10.1242/dmm.050114. Epub 2023 Oct 9.
Cardiomyopathy is often fatal in Friedreich ataxia (FA). However, FA hearts maintain adequate function until advanced disease stages, suggesting initial adaptation to the loss of frataxin (FXN). Conditional cardiac knockout mouse models of FXN show transcriptional and metabolic profiles of the mitochondrial integrated stress response (ISRmt), which could play an adaptive role. However, the ISRmt has not been investigated in models with disease-relevant, partial decrease in FXN. We characterized the heart transcriptomes and metabolomes of three mouse models with varying degrees of FXN depletion: YG8-800, KIKO-700 and FXNG127V. Few metabolites were changed in YG8-800 mice, which did not provide a signature of cardiomyopathy or ISRmt; several metabolites were altered in FXNG127V and KIKO-700 hearts. Transcriptional changes were found in all models, but differentially expressed genes consistent with cardiomyopathy and ISRmt were only identified in FXNG127V hearts. However, these changes were surprisingly mild even at advanced age (18 months), despite a severe decrease in FXN levels to 1% of those of wild type. These findings indicate that the mouse heart has low reliance on FXN, highlighting the difficulty in modeling genetically relevant FA cardiomyopathy.
肥厚型心肌病在弗里德里希共济失调(FA)中通常是致命的。然而,FA 心脏在疾病晚期仍保持足够的功能,这表明其对 frataxin(FXN)缺失有初步的适应能力。FXN 条件性心脏敲除小鼠模型显示出线粒体整合应激反应(ISRmt)的转录和代谢特征,这可能发挥了适应性作用。然而,在具有与疾病相关的 FXN 部分减少的模型中,尚未研究 ISRmt。我们对三种具有不同程度 FXN 耗竭的小鼠模型进行了心脏转录组和代谢组学特征分析:YG8-800、KIKO-700 和 FXNG127V。YG8-800 小鼠的代谢物变化很少,这不能提供心肌病或 ISRmt 的特征;FXNG127V 和 KIKO-700 心脏的几种代谢物发生了改变。所有模型都发现了转录变化,但只有在 FXNG127V 心脏中鉴定到与心肌病和 ISRmt 一致的差异表达基因。然而,即使在老年(18 个月)时,FXN 水平下降到野生型的 1%,这些变化也出人意料地轻微。这些发现表明,小鼠心脏对 FXN 的依赖程度较低,突出了在模型中模拟具有遗传相关性的 FA 心肌病的困难。