Department of Nephrology and Medical Intensive Care, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Leibniz-Forschungsinstitut für Molekulare Pharmakologie (FMP) and Max-Delbrück-Centrum für Molekulare Medizin (MDC), Berlin, Germany.
J Clin Invest. 2023 Feb 1;133(3):e161849. doi: 10.1172/JCI161849.
Sulfate plays a pivotal role in numerous physiological processes in the human body, including bone and cartilage health. A role of the anion transporter SLC26A1 (Sat1) for sulfate reabsorption in the kidney is supported by the observation of hyposulfatemia and hypersulfaturia in Slc26a1-knockout mice. The impact of SLC26A1 on sulfate homeostasis in humans remains to be defined. By combining clinical genetics, functional expression assays, and population exome analysis, we identify SLC26A1 as a sulfate transporter in humans and experimentally validate several loss-of-function alleles. Whole-exome sequencing from a patient presenting with painful perichondritis, hyposulfatemia, and renal sulfate wasting revealed a homozygous mutation in SLC26A1, which has not been previously described to the best of our knowledge. Whole-exome data analysis of more than 5,000 individuals confirmed that rare, putatively damaging SCL26A1 variants were significantly associated with lower plasma sulfate at the population level. Functional expression assays confirmed a substantial reduction in sulfate transport for the SLC26A1 mutation of our patient, which we consider to be novel, as well as for the additional variants detected in the population study. In conclusion, combined evidence from 3 complementary approaches supports SLC26A1 activity as a major determinant of sulfate homeostasis in humans. In view of recent evidence linking sulfate homeostasis with back pain and intervertebral disc disorder, our study identifies SLC26A1 as a potential target for modulation of musculoskeletal health.
硫酸盐在人体的许多生理过程中起着关键作用,包括骨骼和软骨的健康。阴离子转运蛋白 SLC26A1(Sat1)在肾脏中对硫酸盐的重吸收作用得到了 Slc26a1 敲除小鼠低硫酸盐血症和高硫酸盐尿症的观察结果的支持。SLC26A1 对人类硫酸盐稳态的影响仍有待确定。通过结合临床遗传学、功能表达测定和人群外显子组分析,我们确定 SLC26A1 是人类硫酸盐转运体,并实验验证了几个功能丧失等位基因。一名患有软骨膜炎、低硫酸盐血症和肾脏硫酸盐流失的患者的全外显子组测序显示 SLC26A1 存在纯合突变,据我们所知,该突变以前尚未被描述过。对超过 5000 名个体的全外显子组数据进行分析证实,人群水平上罕见的、潜在有害的 SCL26A1 变体与血浆硫酸盐水平显著相关。功能表达测定证实了我们患者的 SLC26A1 突变以及在人群研究中检测到的其他变体的硫酸盐转运能力显著降低,我们认为这是新发现的。总之,3 种互补方法的综合证据支持 SLC26A1 活性是人类硫酸盐稳态的主要决定因素。鉴于最近有证据表明硫酸盐稳态与背痛和椎间盘疾病有关,我们的研究确定 SLC26A1 是调节肌肉骨骼健康的潜在靶点。