The Morris Kahn Laboratory of Human Genetics at the National Institute for Biotechnology in the Negev (NIBN) and Faculty of Health Sciences, Ben Gurion University, Beer-Sheva, Israel.
Hum Mutat. 2013 Apr;34(4):582-6. doi: 10.1002/humu.22274.
Autosomal recessive osteogenesis imperfecta (OI) was diagnosed in three unrelated Israeli Bedouin consanguineous families. Fractures were evident in all cases in infancy. Genome-wide linkage analysis ruled out association with any of the known OI genes, and identified a single homozygosity locus of approximately 2 Mb on chromosome 9 common to all affected individuals (maximum multipoint lod score 6.5). Whole exome sequencing identified only a single mutation within this locus that was shared by all affected individuals: a homozygous deletion mutation of exon 4 of TMEM38B, leading to an early stop codon and a truncated protein, as well as low TMEM38B mRNA levels. TMEM38B encodes TRIC-B, a ubiquitous component of TRIC, a monovalent cation-specific channel involved in Ca(2+) release from intracellular stores that has been shown to act in cell differentiation. Molecular mechanisms through which a TMEM38B mutation might lead to an OI phenotype are yet to be explored.
常染色体隐性遗传性成骨不全症(OI)在三个无关联的以色列贝都因近亲家庭中被诊断出来。所有病例在婴儿期都有明显的骨折。全基因组连锁分析排除了与任何已知的 OI 基因的关联,并确定了一个位于 9 号染色体上的约 2Mb 的单一纯合子位点,所有受影响的个体都存在该位点(最大多点 lod 评分 6.5)。外显子组测序仅在该位点发现了一个突变,所有受影响的个体都携带该突变:TMEM38B 外显子 4 的纯合缺失突变,导致提前出现终止密码子和截短的蛋白质,以及 TMEM38B mRNA 水平降低。TMEM38B 编码 TRIC-B,TRIC 的一种普遍成分,TRIC 是一种单价阳离子特异性通道,参与细胞内储存的 Ca(2+)释放,已被证明在细胞分化中起作用。TMEM38B 突变导致 OI 表型的分子机制尚待探索。