Vitale E, Specchia C, Devoto M, Angius A, Rong S, Rocchi M, Schwalb M, Demelas L, Paglietti D, Manca S, Mastropaolo C, Serra G
Department of Microbiology and Molecular Genetics, UMDNJ New Jersey Medical School, Newark, New Jersey 07103-2714, USA.
Am J Med Genet. 2001 Sep 15;103(1):1-8. doi: 10.1002/ajmg.1495.
We describe a large family from Sardinia, Italy, in which a novel X- linked mental retardation (XLMR) syndrome segregates. The phenotype observed in the 8 affected males includes severe mental retardation (MR), lack of speech, coarse face, distinctive skeletal features with short stature, brachydactyly of fingers and toes, small downslanting palpebral fissures, large bulbous nose, hypoplastic ear lobe and macrostomia. Carrier females are not mentally retarded, although some of them have mild dysmorphic features such as minor ear lobe abnormalities, as well as language and learning problems. Linkage analysis for X-chromosome markers resulted in a maximum lod score of 3.61 with marker DXS1001 in Xq24. Recombination observed with flanking markers identified a region of 16 cM for further study. None of the other XLMR syndromes known to map in the same region shows the same composite phenotype. This evidence strongly suggests that the genetic disease in this family is unique.
我们描述了一个来自意大利撒丁岛的大家族,其中一种新型的X连锁智力迟钝(XLMR)综合征呈分离状态。在8名受影响男性中观察到的表型包括严重智力迟钝(MR)、无言语能力、面容粗糙、具有身材矮小、手指和脚趾短指畸形、小的下斜睑裂、大的球根状鼻、发育不全的耳垂和大口症等独特的骨骼特征。携带者女性智力正常,尽管其中一些人有轻微的畸形特征,如轻微的耳垂异常,以及语言和学习问题。对X染色体标记进行连锁分析,在Xq24区域的标记DXS1001处获得了最高lod分数3.61。与侧翼标记观察到的重组确定了一个16厘摩的区域以供进一步研究。已知定位在同一区域的其他XLMR综合征均未表现出相同的复合表型。这一证据强烈表明该家族中的遗传疾病是独特的。