Moore S W
Division of Pediatric Surgery, Department of Surgical Sciences, Faculty of Health Sciences, University of Stellenbosch, P.O. Box 19063, 7505, Tygerberg, South Africa.
Pediatr Surg Int. 2006 Apr;22(4):305-15. doi: 10.1007/s00383-006-1655-2. Epub 2006 Mar 4.
Hirschsprung's disease (HSCR) is a complex congenital disorder which, from a molecular perspective, appears to result due to disruption of normal signalling during development of enteric nerve cells, resulting in aganglionosis of the distal bowel. Associated congenital anomalies occur in at least 5-32% (mean 21%) of patients and certain syndromic phenotypes have been linked to distinct genetic sites, indicating underlying genetic associations of the disease and probable gene-gene interaction in its pathogenesis. Clear-cut associations with HSCR include Down's syndrome, dominant sensorineural deafness, Waardenburg syndrome, neurofibromatosis, neuroblastoma, phaeochromocytoma, the MEN type IIB syndrome and other abnormalities. Individual anomalies vary from 2.97% to 8%, the most frequent being the gastrointestinal tract (GIT) (8.05%), the central nervous system (CNS) and sensorineural anomalies (6.79%) and the genito-urinary tract (6.05%). Other associated systems include the musculoskeletal (5.12%), cardiovascular systems (4.99%), craniofacial and eye abnormalities (3%) and less frequently the skin and integumentary system (ectodermal dysplasia) and syndromes related to cholesterol and fat metabolism. In addition to associations with neuroblastoma and tumours related to MEN2B, HSCR may also be associated with tumours of neural origin such as ganglioneuroma, ganglioneuroblastoma, retinoblastoma and tumours associated with neurofibromatosis and other autonomic nervous system disturbances. The contribution of the major susceptibility genes on chromosome 10 (RET) and chromosome 13 (EDNRB) is well established in the phenotypic expression of HSCR. Whereas major RET mutations may result in HSCR by haploinsufficiency in 20-25% of cases, the etiology of the majority of sporadic HSCR is not as clear, appearing to arise from the combined cumulative effects of susceptibility loci at critical genes controlling the mechanisms of cell proliferation, differentiation and maturation. In addition, potential "modifying" associations exist with chromosome 2, 9, 20, 21 and 22, and we explore the importance of certain flanking genes of critical areas in the final phenotypic expression of HSCR.
先天性巨结肠症(HSCR)是一种复杂的先天性疾病,从分子角度来看,它似乎是由于肠神经细胞发育过程中正常信号传导受到干扰,导致远端肠道无神经节形成。至少5%-32%(平均21%)的患者会出现相关先天性异常,某些综合征型表型与特定的基因位点相关,这表明该疾病存在潜在的遗传关联以及发病机制中可能的基因-基因相互作用。与HSCR明确相关的疾病包括唐氏综合征、显性遗传性神经性耳聋、瓦登伯革氏综合征、神经纤维瘤病、神经母细胞瘤、嗜铬细胞瘤、MEN IIB型综合征及其他异常。个体异常的发生率在2.97%至8%之间,最常见的是胃肠道(GIT)(8.05%)、中枢神经系统(CNS)和感觉神经性异常(6.79%)以及泌尿生殖系统(6.05%)。其他相关系统包括肌肉骨骼系统(5.12%)、心血管系统(4.99%)、颅面和眼部异常(3%),皮肤和被皮系统(外胚层发育不良)以及与胆固醇和脂肪代谢相关的综合征则较少见。除了与神经母细胞瘤和MEN2B相关的肿瘤有关外,HSCR还可能与神经源性肿瘤如神经节瘤、神经节神经母细胞瘤、视网膜母细胞瘤以及与神经纤维瘤病和其他自主神经系统紊乱相关的肿瘤有关。10号染色体(RET)和13号染色体(EDNRB)上主要易感基因在HSCR表型表达中的作用已得到充分证实。虽然在20%-25%的病例中,主要的RET突变可能通过单倍体不足导致HSCR,但大多数散发性HSCR的病因尚不清楚,似乎是由控制细胞增殖、分化和成熟机制的关键基因处的易感基因座的综合累积效应引起的。此外,2号、9号,20号、21号和22号染色体存在潜在的“修饰”关联,我们探讨了关键区域某些侧翼基因在HSCR最终表型表达中的重要性。