Phenylketonuria (PKU) is an important error of amino acid metabolism which results in most patients from phenylalanine hydroxylase (PAH) deficiency. PKU displays a marked genotypic heterogeneity both within and between different populations. The aim of this study was to establish the genotypic spectrum of PKU in eastern Germany, and to compare this to the distribution of mutations in western Germany. The study population included 302 patients in 290 families who were followed at treatment centers in Berlin, Leipzig and Jena. The study showed marked genotypic variability with a total of 75 mutations, including 15 that have so far not been described (eleven missense mutations, one splicing mutation, and three small deletions). One of these novel mutations, E183Q, occurred in cis to a R408W mutation. In the non-immigrant eastern German population, the frequency of R408W accounted for 40.1% of the PKU alleles. In the immigrant Turkish population of the former West Berlin, the most prevalent mutation was IVS10-11G>A (57%). There was a marked difference of the genotypic spectrum between the population studied here and the data reported from the western part of the country.

译文

:苯丙酮尿症(PKU)是氨基酸代谢的重要错误,导致大多数患者患有苯丙氨酸羟化酶(PAH)缺乏症。北大在不同人群之内和之间都显示出明显的基因型异质性。这项研究的目的是在德国东部建立PKU的基因型谱,并将其与德国西部的突变分布进行比较。研究人群包括290个家庭的302名患者,并在柏林,莱比锡和耶拿的治疗中心进行了随访。这项研究显示出明显的基因型变异,共有75个突变,包括迄今尚未描述的15个突变(11个错义突变,1个剪接突变和3个小缺失)。这些新突变之一E183Q顺式出现在R408W突变中。在非移民的德国东部人口中,R408W的频率占北卡罗来纳等位基因的40.1%。在前西柏林的土耳其移民人口中,最普遍的突变是IVS10-11G> A(57%)。在此研究的人群与该国西部报道的数据之间,基因型谱存在显着差异。

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