Edward Deepak, Al Rajhi Ali, Lewis Richard Alan, Curry Stacey, Wang Zongren, Bejjani Bassem
Department of Ophthalmology and Visual Sciences and Pathology, University of Illinois College of Medicine, Chicago, USA.
Ophthalmic Genet. 2004 Dec;25(4):257-70. doi: 10.1080/13816810490902648.
Peters anomaly (PA) and primary congenital glaucoma (PCG) are genetically and phenotypically distinct conditions. Mutations in cytochrome P4501B1 (CYP1B1) are the most common cause of PCG in Saudi Arabia. Recent evidence suggests that there may be common genetic factors to these conditions. To determine the molecular basis of PA, 11 study subjects with PA from 10 Saudi Arabian families were recruited. Experienced ophthalmologists examined all affected subjects and most of their available unaffected relatives. The diagnosis of PA was confirmed by pathological examination of excised corneal buttons in seven subjects. The coding exons of FOXC1, PITX2, and PAX6 were screened and those of CYP1B1 and FOXE3 sequenced. Homozygous CYP1B1 mutations were identified in six individuals in five families. Five individuals were homozygous for G61E and one was homozygous for 143del10. No mutations were identified in FOXC1, PITX2, PAX6, or FOXE3. The clinical or pathologic phenotype of the subjects with CYP1B1 mutations was not different from that of the other PA patients in this study. Two families included at least one individual with homozygous CYP1B1 mutations and no ocular anomalies (nonpenetrant). Mutations in CYP1B1 may be a substantive cause for PA in this population. Thus, PA and PCG may share a common molecular pathophysiology. Indeed, PA and PCG may share the same spectrum of anterior segment dysgenesis. Finally, the occurrence of PA, PCG, and unaffected individuals with identical homozygous CYP1B1 mutations in the same sibship suggests the presence of modifiers that modulate the clinical severity of the phenotypic expression of the same CYP1B1 mutation(s).
彼得斯异常(PA)和原发性先天性青光眼(PCG)在遗传和表型上是不同的病症。细胞色素P4501B1(CYP1B1)突变是沙特阿拉伯PCG最常见的病因。最近的证据表明,这些病症可能存在共同的遗传因素。为了确定PA的分子基础,招募了来自10个沙特阿拉伯家庭的11名患有PA的研究对象。经验丰富的眼科医生检查了所有受影响的对象及其大多数可获得的未受影响的亲属。通过对7名对象切除的角膜组织块进行病理检查,证实了PA的诊断。对FOXC1、PITX2和PAX6的编码外显子进行了筛查,并对CYP1B1和FOXE3进行了测序。在5个家庭的6名个体中鉴定出纯合的CYP1B1突变。5名个体为G61E纯合子,1名个体为143del10纯合子。在FOXC1、PITX2、PAX6或FOXE3中未鉴定出突变。本研究中,具有CYP1B1突变的对象的临床或病理表型与其他PA患者无异。两个家庭中至少有一名个体具有纯合的CYP1B1突变且无眼部异常(非穿透性)。CYP1B1突变可能是该人群中PA的一个重要病因。因此,PA和PCG可能具有共同的分子病理生理学。事实上,PA和PCG可能具有相同范围的眼前段发育异常。最后,在同一家庭中出现PA、PCG以及具有相同纯合CYP1B1突变的未受影响个体,提示存在调节相同CYP1B1突变表型表达临床严重程度的修饰基因。