Sgariglia Federica, Pedrini Elena, Bradfield Jonathan P, Bhatti Tricia R, D'Adamo Pio, Dormans John P, Gunawardena Aruni T, Hakonarson Hakon, Hecht Jacqueline T, Sangiorgi Luca, Pacifici Maurizio, Enomoto-Iwamoto Motomi, Grant Struan F A
Division of Orthopedic Surgery, Department of Surgery, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Department of Medical Genetics and Skeletal Rare Diseases, IRCCS Rizzoli Orthopaedic Institute (IOR), Bologna, Italy.
Bone. 2015 Mar;72:123-7. doi: 10.1016/j.bone.2014.11.024. Epub 2014 Dec 9.
Hereditary Multiple Exostoses (HME) is an autosomal-dominant disorder characterized by benign cartilage tumors (exostoses) forming near the growth plates, leading to severe health problems. EXT1 and EXT2 are the two genes known to harbor heterozygous loss-of-function mutations that account for the vast majority of the primary genetic component of HME. However, patients present with wide clinical heterogeneity, suggesting that modifier genes play a role in determining severity. Our previous work has pointed to an imbalance of β-catenin signaling being involved in the pathogenesis of osteochondroma formation. TCF7L2 is one of the key 'gate-keeper' TCF family members for Wnt/β-catenin signaling pathway, and TCF7L2 and EXT2 are among the earliest associated loci reported in genome wide appraisals of type 2 diabetes (T2D). Thus we investigated if the key T allele of single nucleotide polymorphism (SNP) rs7903146 within the TCF7L2 locus, which is strongly over-represented among T2D cases, was also associated with HME. We leveraged genotype data available from ongoing GWAS efforts from genomics and orthopedic centers in the US, Canada and Italy. Collectively 213 cases and 1890 controls were analyzed and, surprisingly, the T allele was in fact significantly under-represented in the HME patient group [P = 0.009; odds ratio = 0.737 (95% C.I. 0.587-0.926)]; in addition, the direction of effect was consistent within each individual cohort. Immunohistochemical analyses revealed that TCF7L2 is differentially expressed and distributed in normal human growth plate zones, and exhibits substantial variability in human exostoses in terms of staining intensity and distribution. In summary, the data indicate that there is a putative genetic connection between TCF7L2 and EXT in the context of HME. Given this observation, we suggest that these loci could possibly modulate shared pathways, in particular with respect to β-catenin, and their respective variants interplay to influence HME pathogenesis as well as T2D.
遗传性多发性骨软骨瘤(HME)是一种常染色体显性疾病,其特征是在生长板附近形成良性软骨肿瘤(骨软骨瘤),从而导致严重的健康问题。EXT1和EXT2是已知携带杂合性功能丧失突变的两个基因,这些突变占HME主要遗传成分的绝大部分。然而,患者表现出广泛的临床异质性,这表明修饰基因在决定严重程度方面发挥作用。我们之前的研究指出,β-连环蛋白信号失衡参与了骨软骨瘤形成的发病机制。TCF7L2是Wnt/β-连环蛋白信号通路关键的“守门人”TCF家族成员之一,并且在2型糖尿病(T2D)的全基因组评估中,TCF7L2和EXT2是最早报道的相关基因座。因此,我们研究了TCF7L2基因座内单核苷酸多态性(SNP)rs7903146的关键T等位基因(在T2D病例中强烈过度表达)是否也与HME相关。我们利用了美国、加拿大和意大利的基因组学和骨科中心正在进行的全基因组关联研究(GWAS)中的基因型数据。总共分析了213例病例和1890例对照,令人惊讶的是,T等位基因在HME患者组中实际上显著低于预期[P = 0.009;优势比 = 0.737(95%置信区间0.587 - 0.926)];此外,每个单独队列中的效应方向是一致的。免疫组织化学分析表明,TCF7L2在正常人类生长板区域差异表达和分布,并且在人类骨软骨瘤中在染色强度和分布方面表现出很大的变异性。总之,数据表明在HME背景下TCF7L2和EXT之间存在假定的遗传联系。鉴于这一观察结果,我们认为这些基因座可能调节共享途径,特别是关于β-连环蛋白的途径,并且它们各自的变体相互作用以影响HME发病机制以及T2D。