Center of Excellence in Medical Genetics Research, Chiang Mai University, Chiang Mai, Thailand.
Division of Pediatric Dentistry, Department of Orthodontics and Pediatric Dentistry, Faculty of Dentistry, Chiang Mai University, Chiang Mai, Thailand.
Am J Med Genet A. 2019 Mar;179(3):486-493. doi: 10.1002/ajmg.a.61034. Epub 2019 Jan 17.
Mucopolysaccharidosis Type VII (MPS7, also called β-glucuronidase deficiency or Sly syndrome; MIM 253220) is an extremely rare autosomal recessive lysosomal storage disease, caused by mutations in the GUSB gene. β-glucuronidase (GUSB) is a lysosomal hydrolase involved in the stepwise degradation of glucuronic acid-containing glycosaminoglycans (GAGs). Patients affected with MPS VII are not able to completely degrade glucuronic acid-containing GAGs, including chondroitin 4-sulfate, chondroitin 6-sulfate, dermatan sulfate, and heparan sulfate. The accumulation of these GAGs in lysosomes of various tissues leads to cellular and organ dysfunctions. Characteristic features of MPS VII include short stature, macrocephaly, hirsutism, coarse facies, hearing loss, cloudy cornea, short neck, valvular cardiac defects, hepatosplenomegaly, and dysostosis multiplex. Oral manifestations in patients affected with MPS VII have never been reported. Oral manifestations observed in three patients consist of wide root canal spaces, taurodontism, hyperplastic dental follicles, malposition of unerupted permanent molars, and failure of tooth eruption with malformed roots. The unusual skeletal features of the patients include maxillary hypoplasia, hypoplastic midface, long mandibular length, mandibular prognathism, hypoplastic and aplastic mandibular condyles, absence of the dens of the second cervical vertebra, and erosion of the cortex of the lower border of mandibles. Dogs affected with MPS VII had anterior and posterior open bite, maxillary hypoplasia, premolar crowding, and mandibular prognathism. Unlike patients with MPS VII, the dogs had unremarkable mandibular condyles. This is the first report of oral manifestations in patients affected with MPS VII.
黏多糖贮积症 VII 型(MPS7,也称为β-葡萄糖醛酸酶缺乏症或 Sly 综合征;MIM 253220)是一种极其罕见的常染色体隐性溶酶体贮积症,由 GUSB 基因突变引起。β-葡萄糖醛酸酶(GUSB)是一种溶酶体水解酶,参与含葡萄糖醛酸的糖胺聚糖(GAGs)的逐步降解。患有 MPS7 的患者无法完全降解含葡萄糖醛酸的 GAGs,包括硫酸软骨素 4-硫酸盐、硫酸软骨素 6-硫酸盐、硫酸皮肤素和硫酸乙酰肝素。这些 GAGs 在各种组织的溶酶体中的积累导致细胞和器官功能障碍。MPS7 的特征包括身材矮小、大头畸形、多毛症、粗颜、听力损失、角膜混浊、短颈、瓣膜性心脏缺陷、肝脾肿大和多发性骨发育不良。患有 MPS7 的患者的口腔表现从未有过报道。在 3 名患者中观察到的口腔表现包括根管空间增宽、尖牙变形、牙滤泡增生、未萌出的恒牙位置不正以及牙齿萌出失败伴畸形根。患者的异常骨骼特征包括上颌骨发育不良、中面部发育不良、下颌骨长度长、下颌前突、下颌骨髁突发育不良和发育不全、第二颈椎齿状突缺失以及下颌骨下缘皮质侵蚀。患有 MPS7 的狗出现前牙和后牙开颌、上颌骨发育不良、前磨牙拥挤和下颌前突。与 MPS7 患者不同,这些狗的下颌骨髁突没有明显异常。这是 MPS7 患者口腔表现的首次报告。