Departments of Psychiatry & Behavioral Sciences and Pediatrics, University of Kansas Medical Center, Kansas City, Kansas.
Department of Pharmacology, Toxicology, & Therapeutics, University of Kansas Medical Center, Kansas City, Kansas.
Am J Med Genet A. 2018 Dec;176(12):2587-2594. doi: 10.1002/ajmg.a.40526. Epub 2018 Oct 5.
Prader-Willi syndrome (PWS) is a complex multisystem disorder because of errors in genomic imprinting with severe hypotonia, decreased muscle mass, poor suckling, feeding problems and failure to thrive during infancy, growth and other hormone deficiency, childhood-onset hyperphagia, and subsequent obesity. Decreased energy expenditure in PWS is thought to contribute to reduced muscle mass and physical activity but may also relate to cellular metabolism and disturbances in mitochondrial function. We established fibroblast cell lines from six children and adults with PWS and six healthy controls for mitochondrial assays. We used Agilent Seahorse XF extracellular flux technology to determine real-time measurements of several metabolic parameters including cellular substrate utilization, Adenosine Triphosphate (ATP)-linked respiration, and mitochondrial capacity in living cells. Decreased mitochondrial function was observed in the PWS patients compared to the healthy controls with significant differences in basal respiration, maximal respiratory capacity, and ATP-linked respiration. These results suggest disturbed mitochondrial bioenergetics in PWS although the low number of studied subjects will require a larger subject population before a general consensus can be reached to identify if mitochondrial dysfunction is a contributing factor in PWS.
普拉德-威利综合征(PWS)是一种复杂的多系统疾病,由于基因组印记错误,导致严重的低张力、肌肉量减少、吸吮能力差、喂养问题以及婴儿期、生长和其他激素缺乏时的生长不良,儿童期开始出现暴食,随后出现肥胖。PWS 中的能量消耗减少被认为是导致肌肉减少和身体活动减少的原因,但也可能与细胞代谢和线粒体功能障碍有关。我们从 6 名患有 PWS 的儿童和成人以及 6 名健康对照者中建立了成纤维细胞系,用于进行线粒体检测。我们使用安捷伦 Seahorse XF 细胞外通量技术来实时测定几种代谢参数,包括细胞基质利用、三磷酸腺苷 (ATP) 连接呼吸和活细胞中的线粒体容量。与健康对照组相比,PWS 患者的线粒体功能下降,基础呼吸、最大呼吸能力和 ATP 连接呼吸均有显著差异。这些结果表明 PWS 中存在线粒体生物能量学紊乱,尽管研究对象数量较少,但需要更大的研究对象群体才能得出普遍共识,以确定线粒体功能障碍是否是 PWS 的一个促成因素。