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五例新的脯氨酰寡肽酶缺乏症患者的突变分析:酶活性缺乏导致培养的成纤维细胞出现坏死样细胞死亡。

Mutation analysis of five new patients affected by prolidase deficiency: the lack of enzyme activity causes necrosis-like cell death in cultured fibroblasts.

作者信息

Forlino Antonella, Lupi Anna, Vaghi Patrizia, Icaro Cornaglia Antonia, Calligaro Alberto, Campari Elena, Cetta Giuseppe

机构信息

Dipartimento di Biochimica A. Castellani, University of Pavia, Via Taramelli 3/B, 27100 Pavia, Italy.

出版信息

Hum Genet. 2002 Oct;111(4-5):314-22. doi: 10.1007/s00439-002-0792-5. Epub 2002 Aug 14.

Abstract

Prolidase, a ubiquitously distributed dipeptidase, is involved in the latter stage of degradation of endogenous and dietary proteins and is particularly important in collagen catabolism. It hydrolyzes dipeptides containing proline or hydroxyproline at the C-terminal position. Mutations in the gene encoding for prolidase cause prolidase deficiency (PD), an autosomal recessive disorder mainly characterized by skin lesions, mental retardation and recurrent infectious. In this work we reported the identification of the molecular defect in five PD patients. Direct sequencing of PCR amplified genomic DNA showed a homozygous G>A transversion in two siblings leading to a G448R substitution. A heterozygous IVS11+1G>C transition causing the skipping of exon 11 and a null allele were detected in a third proband. In two unrelated patients, a homozygous IVS7-1G>A transversion was identified and shown to cause multiple alternative spliced transcripts. All the mutations result in loss of prolidase activity. Long-term cultured fibroblasts from these PD patients were used to develop an in vitro model that allowed investigation of the affected cells. Light and electron microscopy revealed that PD cells were more round and branched out than controls with increased cytosolic vacuolization, interruptions of the plasma membrane, mitochondria swelling, mitochondrial matrix and cristae modifications. JC-1 labeling showed decreased mitochondrial membrane potential. A significant intracellular accumulation of the Gly-Pro dipeptide was detected by capillary electrophoresis analysis. Our results provide the first evidence that absence of prolidase activity causes the activation of a necrosis-like cellular death, which could be responsible for the typical skin lesions in PD.

摘要

脯氨酰二肽酶是一种广泛分布的二肽酶,参与内源性和膳食蛋白质降解的后期阶段,在胶原蛋白分解代谢中尤为重要。它水解在C末端位置含有脯氨酸或羟脯氨酸的二肽。编码脯氨酰二肽酶的基因突变会导致脯氨酰二肽酶缺乏症(PD),这是一种常染色体隐性疾病,主要特征为皮肤损伤、智力迟钝和反复感染。在这项研究中,我们报告了5例PD患者分子缺陷的鉴定结果。对PCR扩增的基因组DNA进行直接测序显示,两名同胞中存在纯合的G>A颠换,导致G448R替代。在第三名先证者中检测到杂合的IVS11+1G>C转换,导致外显子11跳跃和一个无效等位基因。在两名无关患者中,鉴定出纯合的IVS7-1G>A颠换,并显示其导致多个可变剪接转录本。所有这些突变均导致脯氨酰二肽酶活性丧失。来自这些PD患者的长期培养成纤维细胞被用于建立一个体外模型,以便对受影响的细胞进行研究。光学显微镜和电子显微镜显示,与对照相比,PD细胞更圆且分支更多,伴有胞质空泡化增加、质膜中断、线粒体肿胀、线粒体基质和嵴的改变。JC-1标记显示线粒体膜电位降低。通过毛细管电泳分析检测到甘氨酰-脯氨酸二肽在细胞内显著积累。我们的结果首次证明,脯氨酰二肽酶活性的缺失会导致类似坏死的细胞死亡激活,这可能是PD中典型皮肤损伤的原因。

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