Prakash Sindhuri, Steers Nicholas J, Li Yifu, Sanchez-Rodriguez Elena, Verbitsky Miguel, Robbins Isabel, Simpson Jenna, Pathak Sharvari, Raska Milan, Reily Colin, Ng Anna, Liang Judy, DeMaria Natalia, Katiraei Amanda, Stevens Kelsey O, Fischman Clara, Shapiro Samantha, Kodali Swetha, McCutchan Jason, Park Heekuk, Eliby Djamila, Delsante Marco, Allegri Landino, Fiaccadori Enrico, Bodria Monica, Marasa Maddalena, Raveche Elizabeth, Julian Bruce A, Uhlemann Anne-Catrin, Kiryluk Krzysztof, Zhang Hong, D'Agati Vivette D, Sanna-Cherchi Simone, Novak Jan, Gharavi Ali G
Department of Medicine, Division of Nephrology, Columbia University Irving Medical Center, College of Physicians and Surgeons, New York, New York, USA.
Department of Microbiology, and.
J Clin Invest. 2025 Mar 18;135(10). doi: 10.1172/JCI181164. eCollection 2025 May 15.
Aberrant O-glycosylation of the IgA1 hinge region is a characteristic finding in patients with IgA nephropathy (IgAN) and is thought to contribute to immune-complex formation and kidney injury. Other studies have suggested that abnormalities in mucosal immunity and lymphocyte homing are major contributors to disease. We identified a family with IgAN segregating a heterozygous predicted loss-of-function (LOF) variant in GALNT14, the gene encoding N-acetylgalactosaminyltransferase 14, one of the enzymes involved in mucin-type protein O-glycosylation. While GALNT14 is expressed in IgA1-producing cells, carriers of the LOF variant did not have altered levels of poorly glycosylated IgA1, suggesting other disease mechanisms. Investigation of Galnt14-null mice revealed elevated serum IgA levels and ex vivo IgA production by B cells. These mice developed glomerular IgA deposition with aging and after induction of sterile colitis. Galnt14-null mice also displayed an attenuated mucin layer in the colon and redistribution of IgA-producing cells from mucosal to systemic sites. Adoptive-transfer experiments indicated impaired homing of spleen-derived Galnt14-deficient B lymphocytes, resulting in increased retention in peripheral blood. These findings suggest that abnormalities in O-glycosylation alter mucosal immunity and B lymphocyte homing, pointing to an expanded role of aberrant O-glycosylation in the pathogenesis of IgAN.
IgA1铰链区异常O-糖基化是IgA肾病(IgAN)患者的一个特征性发现,被认为与免疫复合物形成和肾损伤有关。其他研究表明,黏膜免疫和淋巴细胞归巢异常是该疾病的主要促成因素。我们鉴定了一个患有IgAN的家系,该家系中GALNT14基因存在杂合的预测功能丧失(LOF)变异,GALNT14基因编码N-乙酰半乳糖胺基转移酶14,是参与粘蛋白型蛋白O-糖基化的酶之一。虽然GALNT14在产生IgA1的细胞中表达,但LOF变异的携带者未出现糖基化不足的IgA1水平改变,提示存在其他疾病机制。对Galnt14基因敲除小鼠的研究显示血清IgA水平升高以及B细胞体外产生IgA。这些小鼠随着年龄增长以及在无菌性结肠炎诱导后出现肾小球IgA沉积。Galnt14基因敲除小鼠在结肠中还表现出粘蛋白层变薄以及产生IgA的细胞从黏膜部位重新分布到全身部位。过继转移实验表明脾脏来源的Galnt14缺陷型B淋巴细胞归巢受损,导致在外周血中的滞留增加。这些发现提示O-糖基化异常会改变黏膜免疫和B淋巴细胞归巢,表明异常O-糖基化在IgAN发病机制中的作用扩大。