{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,10]],"date-time":"2026-08-10T14:49:33Z","timestamp":1786373373824,"version":"build-2736575974"},"reference-count":119,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"6","funder":[{"name":"Canadian Institutes of Health Research\/KRESCENT Early Career Investigators in Maternal, Reproductive, Child and Youth Health","award":["2019KP-NIOG01"],"award-info":[{"award-number":["2019KP-NIOG01"]}]}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":["CJASN"],"published-print":{"date-parts":[[2021,6]]},"abstract":"<jats:p>In the past 20 years, we have witnessed tremendous advances in our ability to diagnose and treat genetic diseases of the kidney caused by complement dysregulation. Staggering progress was realized toward a better understanding of the genetic underpinnings and pathophysiology of many forms of atypical hemolytic uremic syndrome (aHUS) and C3-dominant glomerulopathies that are driven by complement system abnormalities. Many of these seminal discoveries paved the way for the design and characterization of several innovative therapies, some of which have already radically improved patients\u2019 outcomes. This review offers a broad overview of the exciting developments that have occurred in the recent past, with a particular focus on single-gene (or Mendelian), complement-driven aHUS and C3-dominant glomerulopathies that should be of interest to both nephrologists and kidney researchers. The discussion is restricted to genes with robust associations with both aHUS and C3-dominant glomerulopathies (complement factor H, complement component 3, complement factor H\u2013related proteins) or only aHUS (complement factor B, complement factor I, and membrane cofactor protein). Key questions and challenges are highlighted, along with potential avenues for future directions.<\/jats:p>","DOI":"10.2215\/cjn.11830720","type":"journal-article","created":{"date-parts":[[2021,2,3]],"date-time":"2021-02-03T15:53:02Z","timestamp":1612367582000},"page":"942-956","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":57,"title":["Inherited Kidney Complement Diseases"],"prefix":"10.2215","volume":"16","author":[{"given":"Mathieu","family":"Lemaire","sequence":"first","affiliation":[{"name":"Division of Nephrology, The Hospital for Sick Children, Toronto, Ontario, Canada"},{"name":"Cell Biology Program, SickKids Research Institute, Toronto, Ontario, Canada"},{"name":"Department of Paediatrics, Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Damien","family":"Noone","sequence":"additional","affiliation":[{"name":"Division of Nephrology, The Hospital for Sick Children, Toronto, Ontario, Canada"},{"name":"Department of Paediatrics, Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne-Laure","family":"Lapeyraque","sequence":"additional","affiliation":[{"name":"Division of Nephrology, Sainte-Justine University Hospital Center, Montreal, Quebec, Canada"},{"name":"Department of Pediatrics, Faculty of Medicine, University of Montr\u00e9al, Qu\u00e9bec, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Licht","sequence":"additional","affiliation":[{"name":"Division of Nephrology, The Hospital for Sick Children, Toronto, Ontario, Canada"},{"name":"Cell Biology Program, SickKids Research Institute, Toronto, Ontario, Canada"},{"name":"Department of Paediatrics, Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V\u00e9ronique","family":"Fr\u00e9meaux-Bacchi","sequence":"additional","affiliation":[{"name":"Assistance Publique-H\u00f4pitaux de Paris, H\u00f4pital Europ\u00e9en Georges Pompidou, Laboratory of Immunology, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2021,6]]},"reference":[{"key":"B1-20241015","doi-asserted-by":"crossref","first-page":"814","DOI":"10.1542\/peds.8.6.814","article-title":"Serum complement in acute glomerulonephritis and the nephrotic syndrome","volume":"8","author":"Lange","year":"1951","journal-title":"Pediatrics"},{"key":"B2-20241015","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1146\/annurev.me.19.020168.000245","article-title":"Complement in human disease","volume":"19","author":"Schur","year":"1968","journal-title":"Annu Rev Med"},{"key":"B3-20241015","first-page":"101","article-title":"Membranoproliferative glomerulonephritis in two sibships","volume":"16","author":"Berry","year":"1981","journal-title":"Clin Nephrol"},{"key":"B4-20241015","doi-asserted-by":"crossref","first-page":"681","DOI":"10.1038\/ki.1983.211","article-title":"Inherited complement component deficiencies in membranoproliferative glomerulonephritis","volume":"24","author":"Coleman","year":"1983","journal-title":"Kidney Int"},{"key":"B5-20241015","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1093\/ajcp\/70.3.434","article-title":"Adult hemolytic uremic syndrome with renal arteriolar deposition of IgM andC3","volume":"70","author":"Hammar","year":"1978","journal-title":"Am J Clin Pathol"},{"key":"B6-20241015","doi-asserted-by":"crossref","first-page":"1090","DOI":"10.1056\/NEJM197505222922102","article-title":"Hemolytic uremic syndrome in families","volume":"292","author":"Kaplan","year":"1975","journal-title":"N Engl J Med"},{"key":"B7-20241015","first-page":"110","article-title":"Hypocomplementaemia due to a genetic deficiency of beta 1H globulin","volume":"46","author":"Thompson","year":"1981","journal-title":"Clin Exp Immunol"},{"key":"B8-20241015","doi-asserted-by":"crossref","first-page":"25168","DOI":"10.1074\/jbc.272.40.25168","article-title":"Human factor H deficiency. 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