Carella M, Stewart G, Ajetunmobi J F, Perrotta S, Grootenboer S, Tchernia G, Delaunay J, Totaro A, Zelante L, Gasparini P, Iolascon A
Servizio di Genetica Medica, Istituto di Ricovero e Carattere Scientifico, San Giovanni Rotondo, Italy.
Am J Hum Genet. 1998 Sep;63(3):810-6. doi: 10.1086/302024.
Dehydrated hereditary stomatocytosis, also known as "hereditary xerocytosis," is caused by a red blood cell-membrane defect characterized by stomatocytic morphology, increased mean corpuscular hemoglobin concentration, decreased osmotic fragility, increased permeability to the univalent cations Na+ and K+, and an increased proportion of phosphatidylcholine in the membrane. The clinical presentation is heterogeneous, ranging from mild to moderate hemolytic anemia associated with scleral icterus, splenomegaly, and choletithiasis. Iron overload may develop later in life. The disease is transmitted as an autosomal dominant trait. We recruited a large three-generation Irish family affected with DHS and comprising 23 members, of whom 14 were affected and 9 were healthy. Two additional, small families also were included in the study. The DNA samples from the family members were used in a genomewide search to identify, by linkage analysis, the DHS locus. After the exclusion of a portion of the human genome, we obtained conclusive evidence for linkage of DHS to microsatellite markers on the long arm of chromosome 16 (16q23-q24). A maximum two-point LOD score of 6.62 at recombination fraction .00 was obtained with marker D16S520. There are no recombination events defining the telomeric limit of the region, which therefore is quite large. No candidate genes map to this area.
脱水遗传性口形红细胞增多症,也称为“遗传性干皮症”,由红细胞膜缺陷引起,其特征为口形红细胞形态、平均红细胞血红蛋白浓度增加、渗透脆性降低、对单价阳离子Na⁺和K⁺的通透性增加以及膜中磷脂酰胆碱比例增加。临床表现具有异质性,范围从轻度至中度溶血性贫血,伴有巩膜黄疸、脾肿大和胆结石。铁过载可能在生命后期出现。该疾病以常染色体显性性状遗传。我们招募了一个受脱水遗传性口形红细胞增多症影响的爱尔兰大家庭,由23名成员组成,其中14人患病,9人健康。另外两个小家庭也被纳入研究。家庭成员的DNA样本用于全基因组搜索,通过连锁分析确定脱水遗传性口形红细胞增多症基因座。在排除人类基因组的一部分后,我们获得了脱水遗传性口形红细胞增多症与16号染色体长臂(16q23-q24)上微卫星标记连锁的确凿证据。使用标记D16S520在重组率为0.00时获得的最大两点LOD评分为6.62。没有重组事件定义该区域的端粒界限,因此该区域相当大。没有候选基因定位于此区域。