Ullah Asmat, Umair Muhammad, Muhammad Dost, Bilal Muhammad, Lee Kwanghyuk, Leal Suzanne M, Ahmad Wasim
Department of Biochemistry, Faculty of Biological Sciences, Quaid-i-Azam University, Islamabad, Pakistan.
Chandka Medical College, Shaheed Mohtarma Benazir Bhutto Medical University Larkana, Sindh, Pakistan.
Ann Hum Genet. 2018 May;82(3):129-134. doi: 10.1111/ahg.12233. Epub 2018 Jan 10.
Acromesomelic dysplasia is genetically heterogeneous group of skeletal disorders characterized by short stature and acromelia and mesomelia of limbs. Acromesomelic dysplasia segregates in an autosomal recessive pattern and is caused by biallelic sequence variants in three genes (NPR2, GDF5, and BMPR1B). A consanguineous family of Pakistani origin segregating a subtype of acromesomelic dysplasia called Hunter-Thompson was clinically and genetically evaluated. Genotyping of microsatellite markers and linkage analysis revealed a 7.78 Mb homozygous region on chromosome 4q22.3, which harbors BMPR1B. Sequence analysis of the gene revealed a novel homozygous missense variant (c.1190T > G, p.Met397Arg) that segregates with the disease phenotype within the family and produced a Logarithm of odds (LOD) score of 3.9 with the disease phenotype. This study reports on the first familial case of acromesomelic dysplasia Hunter-Thompson type. It is also the first report of BMPR1B underlying the etiology of acromesomelic dysplasia Hunter-Thompson type.
肢端中胚层发育不良是一组遗传性骨骼疾病,其特征为身材矮小以及四肢的肢端短小和中胚层短小。肢端中胚层发育不良以常染色体隐性模式遗传,由三个基因(NPR2、GDF5和BMPR1B)中的双等位基因序列变异引起。对一个来自巴基斯坦的近亲家庭进行了临床和基因评估,该家庭患有肢端中胚层发育不良的一种亚型,即亨特-汤普森型。微卫星标记的基因分型和连锁分析显示,4号染色体q22.3上有一个7.78兆碱基的纯合区域,该区域包含BMPR1B。对该基因的序列分析发现了一个新的纯合错义变异(c.1190T>G,p.Met397Arg),该变异在家族中与疾病表型共分离,与疾病表型的对数优势(LOD)得分为3.9。本研究报告了首例亨特-汤普森型肢端中胚层发育不良的家族病例。这也是关于BMPR1B是亨特-汤普森型肢端中胚层发育不良病因的首次报告。