Familial nephropathy and multiple exostoses with exostosin-1 (EXT1) gene mutation

ISD Roberts, JM Gleadle - Journal of the American Society of …, 2008 - journals.lww.com
ISD Roberts, JM Gleadle
Journal of the American Society of Nephrology, 2008journals.lww.com
Glomerular deposition of fibrillar collagen is a characteristic finding of genetically distinct
conditions, including nail-patella syndrome and collagen type III glomerulopathy. A case of
familial nephropathy in which steroid-sensitive nephrotic syndrome and glomerular deposits
of fibrillar collagen are associated with multiple exostoses due to mutation of the EXT1 gene
is described. This gene encodes a glycosyltransferase required for synthesis of heparan
sulfate glycosaminoglycans. There is deficiency of heparan sulfate and perlecan, together …
Abstract
Glomerular deposition of fibrillar collagen is a characteristic finding of genetically distinct conditions, including nail-patella syndrome and collagen type III glomerulopathy. A case of familial nephropathy in which steroid-sensitive nephrotic syndrome and glomerular deposits of fibrillar collagen are associated with multiple exostoses due to mutation of the EXT1 gene is described. This gene encodes a glycosyltransferase required for synthesis of heparan sulfate glycosaminoglycans. There is deficiency of heparan sulfate and perlecan, together with accumulation of collagens, in the matrix of EXT1-associated osteochondromas. Similar glomerular basement membrane abnormalities could offer an explanation for both the renal ultrastructural changes and steroid-sensitive nephrotic syndrome.
Lippincott Williams & Wilkins