Dr. John T. Macdonald Foundation Department of Human Genetics and John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL, USA.
Department of Neuromuscular Disease, UCL Queen Square Institute of Neurology and The National Hospital for Neurology, London, UK.
Nat Genet. 2020 May;52(5):473-481. doi: 10.1038/s41588-020-0615-4. Epub 2020 May 4.
Here we report biallelic mutations in the sorbitol dehydrogenase gene (SORD) as the most frequent recessive form of hereditary neuropathy. We identified 45 individuals from 38 families across multiple ancestries carrying the nonsense c.757delG (p.Ala253GlnfsTer27) variant in SORD, in either a homozygous or compound heterozygous state. SORD is an enzyme that converts sorbitol into fructose in the two-step polyol pathway previously implicated in diabetic neuropathy. In patient-derived fibroblasts, we found a complete loss of SORD protein and increased intracellular sorbitol. Furthermore, the serum fasting sorbitol levels in patients were dramatically increased. In Drosophila, loss of SORD orthologs caused synaptic degeneration and progressive motor impairment. Reducing the polyol influx by treatment with aldose reductase inhibitors normalized intracellular sorbitol levels in patient-derived fibroblasts and in Drosophila, and also dramatically ameliorated motor and eye phenotypes. Together, these findings establish a novel and potentially treatable cause of neuropathy and may contribute to a better understanding of the pathophysiology of diabetes.
我们报道了双等位基因突变在山梨醇脱氢酶基因(SORD)作为最常见的遗传性神经病的隐性形式。我们从 38 个家族中发现了 45 个个体,他们携带 SORD 中的无义 c.757delG(p.Ala253GlnfsTer27)变异,无论是纯合子还是复合杂合子状态。SORD 是一种酶,可将山梨醇转化为果糖,该酶在先前与糖尿病神经病相关的两步多元醇途径中起作用。在患者来源的成纤维细胞中,我们发现 SORD 蛋白完全缺失,细胞内山梨醇增加。此外,患者的血清空腹山梨醇水平显著增加。在果蝇中,SORD 同源物的缺失导致突触退化和进行性运动障碍。通过用醛糖还原酶抑制剂治疗减少多元醇的流入,可使患者来源的成纤维细胞和果蝇中的细胞内山梨醇水平正常化,并且还可显著改善运动和眼睛表型。这些发现确立了一种新的、潜在可治疗的神经病原因,并可能有助于更好地理解糖尿病的病理生理学。