Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada, K1H 8L6.
Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Ontario, Canada, K1H 8M5.
Ann Clin Transl Neurol. 2019 Nov;6(11):2340-2346. doi: 10.1002/acn3.50920. Epub 2019 Oct 13.
Spinal muscular atrophy (SMA) is a neuromuscular disorder leading to paralysis and death. Recent evidence shows increased susceptibility to dyslipidemia and liver steatosis in patients. Here, we provide evidence that low fat diets nearly double survival in Smn mice, a model for SMA, independent of changes in SMN levels, liver steatosis, or enhanced hepatic functions. Liver damage and ketone levels were reduced, implying a lower reliance on fatty acid oxidation. This preclinical proof of concept study provides grounds for controlled clinical investigation of dietary needs and offers evidence to inform nutritional guidelines specific to SMA.
脊髓性肌萎缩症（SMA）是一种导致瘫痪和死亡的神经肌肉疾病。最近的证据表明，患者更容易出现血脂异常和肝脂肪变性。在这里，我们提供的证据表明，低脂饮食可使 SMA 模型 Smn 小鼠的存活率几乎增加一倍，这与 SMN 水平、肝脂肪变性或增强的肝功能变化无关。肝损伤和酮体水平降低，意味着对脂肪酸氧化的依赖降低。这项临床前概念验证研究为控制饮食需求的临床研究提供了依据，并为制定针对 SMA 的特定营养指南提供了证据。
