Kövesdi Erzsébet, Bene Judit, Nagy Nikoletta, Horváth Ágnes, Melegh Béla, Hadzsiev Kinga
Klinikai Központ, Orvosi Genetikai Intézet, Pécsi Tudományegyetem, Általános Orvostudományi Kar Pécs, Szigeti út 12., 7624.
Szentágothai János Kutatóközpont, Pécsi Tudományegyetem Pécs.
Orv Hetil. 2017 Jul;158(30):1188-1194. doi: 10.1556/650.2017.30789.
Tuberous sclerosis complex is a rare disease with high phenotypic heterogeneity, characterized by the appearance of multiplex hamartomas in the different organs. The disease is inherited by autosomal dominant manner, due to the mutations of two genes: the TSC1 or the TSC2. In this publication we present the cases of two young male and two middle-aged female patients, where pathogenetic differences of TSC1/TSC2 could not be verified by Sanger sequencing. However, multiplex ligation-dependent probe amplification confirmed different sizes of deletions in different regions of the TSC2 gene. All patients carry the typical clinical signs of the disease. However, the individual phenotypic variability is very different. With this manuscript, we would like to draw attention to the relative frequent rate of gross gene deletions. Orv Hetil. 2017; 158(30): 1188-1194.
结节性硬化症是一种罕见病,具有高度的表型异质性,其特征是在不同器官出现多发性错构瘤。该疾病以常染色体显性方式遗传,由两个基因(TSC1或TSC2)的突变引起。在本出版物中,我们展示了两名年轻男性和两名中年女性患者的病例,其中通过桑格测序无法验证TSC1/TSC2的致病差异。然而,多重连接依赖探针扩增证实了TSC2基因不同区域存在不同大小的缺失。所有患者都具有该疾病的典型临床体征。然而,个体表型变异性差异很大。通过本手稿,我们想提请注意基因大片段缺失的相对频繁发生率。《匈牙利医学周报》。2017年;158(30):1188 - 1194。