Suppr超能文献

肌营养不良蛋白mRNA的可变剪接使携带者的确定变得复杂:一个杜氏肌营养不良症(DMD)家系的报告。

Alternative splicing of dystrophin mRNA complicates carrier determination: report of a DMD family.

作者信息

Lenk U, Demuth S, Kräft U, Hanke R, Speer A

机构信息

Max Delbrück Centre for Molecular Medicine, Berlin-Buch, Germany.

出版信息

J Med Genet. 1993 Mar;30(3):206-9. doi: 10.1136/jmg.30.3.206.

Abstract

Carrier determination is important for genetic counselling in DMD/BMD families. The detection of altered PCR amplified dystrophin mRNA fragments owing to deletions, insertions, or point mutations has increased the possibilities of carrier determination. However, problems may occur because of alternative splicing events. Here we present a family with a DMD patient characterised by a deletion of exons 45 to 54. At the mRNA level we detected a corresponding altered fragment which served for carrier determination. The mother and the sister of the patient showed the same altered dystrophin mRNA fragment as the patient and are therefore carriers. In the mother two additional altered dystrophin mRNA fragments were detectable, obviously resulting from alternative splicing in the normal allele. The grandmother and two other related females of the patient possess only the normal mRNA fragment. In a further female we detected an altered fragment owing to an mRNA deletion of exon 44. This fragment is created either by alternative splicing or a new mutation. Therefore, the carrier status of this female is still ambiguous indicating problems in carrier determination by the method of dystrophin mRNA analysis.

摘要

对于杜氏肌营养不良症(DMD)/贝克型肌营养不良症(BMD)家族的遗传咨询而言,携带者检测至关重要。由于缺失、插入或点突变导致的聚合酶链反应(PCR)扩增的抗肌萎缩蛋白mRNA片段改变的检测,增加了携带者检测的可能性。然而,由于可变剪接事件,可能会出现问题。在此,我们展示一个患有DMD的家族,其特征为外显子45至54缺失。在mRNA水平,我们检测到一个相应的改变片段,可用于携带者检测。患者的母亲和姐姐显示出与患者相同的抗肌萎缩蛋白mRNA片段改变,因此她们是携带者。在母亲体内还可检测到另外两个改变的抗肌萎缩蛋白mRNA片段,显然这是由正常等位基因中的可变剪接产生的。患者的祖母和另外两名相关女性仅拥有正常的mRNA片段。在另一名女性中,我们检测到一个由于外显子44的mRNA缺失而改变的片段。这个片段要么是由可变剪接产生,要么是新的突变所致。因此,该女性的携带者状态仍不明确,这表明通过抗肌萎缩蛋白mRNA分析方法进行携带者检测存在问题。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0a42/1016300/46920a3c805e/jmedgene00005-0035-a.jpg

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验