两个患有2型德斯布瓦氏发育不良的土耳其家庭中的新型和复发性XYLT1突变。

Novel and recurrent XYLT1 mutations in two Turkish families with Desbuquois dysplasia, type 2.

作者信息

Guo Long, Elcioglu Nursel H, Iida Aritoshi, Demirkol Yasemin K, Aras Seda, Matsumoto Naomichi, Nishimura Gen, Miyake Noriko, Ikegawa Shiro

机构信息

Laboratory for Bone and Joint Diseases, RIKEN Center for Integrative Medical Sciences, Tokyo, Japan.

Department of Pediatric Genetics, Marmara University Medical School, Istanbul, Turkey.

出版信息

J Hum Genet. 2017 Mar;62(3):447-451. doi: 10.1038/jhg.2016.143. Epub 2016 Nov 24.

Abstract

Desbuquois dysplasia (DBQD) is an autosomal recessive skeletal disorder characterized by growth retardation, joint laxity, short extremities, and progressive scoliosis. DBQD is classified into two types based on the presence (DBQD1) or absence (DBQD2) of characteristic hand abnormalities. CANT1 mutations have been reported in both DBQD1 and DBQD2. Recently, mutations in the gene encoding xylosyltransferase 1 (XYLT1) were identified in several families with DBQD2. In this study, we performed whole-exome sequencing in two Turkish families with DBQD2. We found a novel and a recurrent XYLT1 mutation in each family. The patients were homozygous for the mutations. Our results further support that XYLT1 is responsible for a major subset of DBQD2.

摘要

德布夸氏发育不全(DBQD)是一种常染色体隐性遗传性骨骼疾病,其特征为生长发育迟缓、关节松弛、四肢短小以及进行性脊柱侧弯。根据特征性手部异常的有无,DBQD分为两型(DBQD1型和DBQD2型)。DBQD1型和DBQD2型均有CANT1基因突变的报道。最近,在几个DBQD2型家族中发现了编码木糖基转移酶1(XYLT1)的基因突变。在本研究中,我们对两个患有DBQD2型的土耳其家族进行了全外显子组测序。我们在每个家族中发现了一个新的和一个复发的XYLT1突变。患者为这些突变的纯合子。我们的结果进一步支持XYLT1是DBQD2型的主要致病原因。

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