Srivastava Priyanka, Tuteja Moni, Dalal Ashwin, Mandal Kausik, R Phadke Shubha
Department of Medical Genetics, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Raebareli Road, Lucknow 226 014, India.
J Genet. 2016 Dec;95(4):905-909. doi: 10.1007/s12041-016-0715-1.
Acromesomelic dysplasia, type Maroteaux is a disorder characterized by disproportionate short stature predominantly affecting the middle and distal segments of the upper and lower limbs. It is an autosomal recessive disorder due to mutation in NPR2 gene which impairs skeletal growth. To screen the mutations in the gene NPR2, all of its coding exons and splice junction sites were PCR amplified from genomic DNA of affected individuals of four families and sequenced. Four homozygous mutations in four different families were identified. These include three novel mutations including a deletion frameshift mutation (p.Cys586Ter), one nonsense mutation (p.Arg479Ter), one missense mutation (p.Val187Asp) and one reported missense mutation (p.Tyr338Cys). The study describes phenotypes of Indian patients and expands the mutation spectrum of the disorder.
马罗泰克斯型肢端中胚层发育不良是一种以身材不成比例矮小为特征的疾病,主要影响上肢和下肢的中段和远端。它是一种常染色体隐性疾病,由NPR2基因突变导致骨骼生长受损。为了筛查NPR2基因的突变,从四个家族的患病个体的基因组DNA中PCR扩增其所有编码外显子和剪接连接位点并进行测序。在四个不同家族中鉴定出四个纯合突变。这些包括三个新突变,其中一个缺失移码突变(p.Cys586Ter)、一个无义突变(p.Arg479Ter)、一个错义突变(p.Val187Asp)和一个已报道的错义突变(p.Tyr338Cys)。该研究描述了印度患者的表型并扩展了该疾病的突变谱。