Méndez M, Morán-Jiménez M J, Gomez-Abecia S, García-Bravo M, Garrido-Astray M C, Fontanellas A, Poblete-Gutiérrez P, Frank J, Enriquez de Salamanca R
Research Center. Hospital Universitario 12 de Octubre, Madrid, Spain.
Cell Mol Biol (Noisy-le-grand). 2009 Jul 1;55(2):55-63.
Acute intermittent porphyria (AIP), the most common acute hepatic porphyria, is an autosomal dominant disorder with low penetrance that results from a partial deficiency of hydroxymethylbilane synthase (HMBS), the third enzyme in the heme biosynthetic pathway. The disease is clinically characterized by acute neurovisceral attacks that are precipitated by several factors including certain drugs, steroid hormones, alcohol and fasting. Early diagnosis and counselling are essential to prevent attacks, being mutation analysis the most reliable method to identify asymptomatic carriers in AIP families. In this study we have investigated the molecular defect in 15 unrelated Spanish AIP patients. Mutation analysis of the HMBS gene revealed a total of fourteen mutations including six novel ones, two of them were on the same allele in one patient. The novel mutations were three missense (R26L, R173G and D178H), two frameshift (c.749_765dup and c.874insC) and one intronic deletion (IVS12+3_+11delAGGGCCTGT). RT-PCR and sequencing demonstrated that the intronic mutation caused abnormal splicing and exon 12 skipping. Prokaryotic expression of the novel missense mutations showed that only D178H had significant residual activity. These findings will facilitate the accurate identification of presymptomatic AIP carriers in these families and they further emphasize the molecular heterogeneity of AIP in Spain.
急性间歇性卟啉病(AIP)是最常见的急性肝性卟啉病,是一种常染色体显性疾病,外显率低,由血红素生物合成途径中的第三种酶——羟甲基胆色素原合酶(HMBS)部分缺乏所致。该疾病的临床特征为急性神经内脏发作,可由多种因素诱发,包括某些药物、类固醇激素、酒精和禁食。早期诊断和咨询对于预防发作至关重要,其中突变分析是识别AIP家族中无症状携带者最可靠的方法。在本研究中,我们调查了15名无亲缘关系的西班牙AIP患者的分子缺陷。对HMBS基因的突变分析共发现14种突变,其中包括6种新突变,其中2种在一名患者的同一等位基因上。新突变包括3种错义突变(R26L、R173G和D178H)、2种移码突变(c.749_765dup和c.874insC)和1种内含子缺失(IVS12 +3_+11delAGGGCCTGT)。RT-PCR和测序表明,内含子突变导致异常剪接和外显子12跳跃。新错义突变的原核表达表明,只有D178H具有显著的残余活性。这些发现将有助于准确识别这些家族中无症状的AIP携带者,并进一步强调西班牙AIP的分子异质性。