Molecular Medicine & Neurobiology-ZebraLab, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy.
Department of Veterinary Science, University of Pisa, 56124 Pisa, Italy.
Nutrients. 2021 Mar 19;13(3):998. doi: 10.3390/nu13030998.
Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin gene, is an inherited neuromuscular disorder that causes loss of muscle mass and motor skills. In the era of genomic medicine, there is still no known cure for DMD. In clinical practice, there is a growing awareness of the possible importance of nutrition in neuromuscular diseases. This is mostly the result of patients' or caregivers' empirical reports of how active substances derived from food have led to improved muscle strength and, thus, better quality of life. In this report, we investigate several nutraceutical principles in the strain of zebrafish, a validated model of DMD, in order to identify possible natural products that, if supplemented in the diet, might improve the quality of life of DMD patients. Gingerol, a constituent of fresh ginger, statistically increased the locomotion of mutant larvae and upregulated the expression of heme oxygenase 1, a target gene for therapy aimed at improving dystrophic symptoms. Although three other compounds showed a partial positive effect on locomotor and muscle structure phenotypes, our nutraceutical screening study lent preliminary support to the efficacy and safety only of gingerol. Gingerol could easily be proposed as a dietary supplement in DMD.
杜氏肌营养不良症(DMD)是一种遗传性神经肌肉疾病,由肌营养不良蛋白基因突变引起,会导致肌肉质量和运动技能丧失。在基因组医学时代,DMD 仍然没有已知的治愈方法。在临床实践中,人们越来越意识到营养在神经肌肉疾病中的可能重要性。这主要是由于患者或护理人员的经验报告表明,源自食物的活性物质如何导致肌肉力量增强,从而提高生活质量。在本报告中,我们研究了几种营养原则在 DMD 验证模型斑马鱼品系中的作用,以确定如果在饮食中补充可能改善 DMD 患者生活质量的天然产物。姜辣素是新鲜生姜的一种成分,它在统计学上增加了突变幼虫的运动能力,并上调了血红素加氧酶 1 的表达,血红素加氧酶 1 是一种旨在改善肌营养不良症状的治疗靶标基因。尽管另外三种化合物对运动和肌肉结构表型有部分积极影响,但我们的营养筛选研究仅初步支持姜辣素的疗效和安全性。姜辣素可以很容易地被提议作为 DMD 的膳食补充剂。