Ehtesham Nadia, Cantor Rita M, King Lily M, Reinker Kent, Powell Berkley R, Shanske Alan, Unger Sheila, Rimoin David L, Cohn Daniel H
Medical Genetics-Birth Defects Center, Ahmanson Department of Pediatrics, Cedars-Sinai Research Institute, Los Angeles, CA 90048, USA.
Am J Hum Genet. 2002 Oct;71(4):947-51. doi: 10.1086/342669. Epub 2002 Aug 2.
Smith-McCort dysplasia is a rare autosomal recessive osteochondrodysplasia characterized by short limbs and a short trunk with a barrel-shaped chest. The radiographic phenotype includes platyspondyly, generalized abnormalities of the epiphyses and metaphyses, and a distinctive lacy appearance of the iliac crest. We performed a genomewide scan in a consanguineous family from Guam and found evidence of linkage to loci on chromosome 18q12. Analysis of a second, smaller family was also consistent with linkage to this region, producing a maximum combined two-point LOD score of 3.04 at a recombination fraction of 0 for the marker at locus D18S450. A 10.7-cM region containing the disease gene was defined by recombination events in two affected individuals in the larger family. Furthermore, all affected children in the larger family were homozygous for a subset of marker loci within this region, defining a 1.5-cM interval likely to contain the defective gene. Analysis of three small, unrelated families with Dyggve-Melchior-Clausen syndrome, a radiographically identical disorder with the additional clinical finding of mental retardation, provided evidence of linkage to the same region, a result consistent with the hypothesis that the two disorders are allelic.
史密斯-麦科特发育异常是一种罕见的常染色体隐性骨软骨发育不良,其特征为四肢短小、躯干短小且胸部呈桶状。影像学表型包括椎体扁平、骨骺和干骺端普遍异常以及髂嵴独特的花边样外观。我们对来自关岛的一个近亲家庭进行了全基因组扫描,发现与18号染色体q12上的位点存在连锁证据。对另一个较小的家庭进行分析也与该区域的连锁一致,在重组率为0时,位点D18S450处的最大两点LOD值为3.04。通过大家庭中两名受影响个体的重组事件确定了一个包含疾病基因的10.7厘摩区域。此外,大家庭中所有受影响的儿童在该区域内的一部分标记位点上是纯合的,确定了一个可能包含缺陷基因的1.5厘摩区间。对三个患有迪格维-梅尔基奥尔-克劳森综合征(一种影像学表现相同但伴有智力发育迟缓这一额外临床发现的疾病)的无关小家庭进行分析,提供了与同一区域连锁的证据,这一结果与两种疾病是等位基因的假设一致。