利用长读序列技术进行单倍型构建和预防常染色体显性多囊肾病在嵌合家族的传播。

IF 2.6
DNA and cell biology Pub Date : 2025-05-01 Epub Date: 2025-04-02 DOI:10.1089/dna.2024.0280
Wu Zubo, Jie Liu, Yi Liu, Xiaoli Wang, Defeng Shu
{"title":"利用长读序列技术进行单倍型构建和预防常染色体显性多囊肾病在嵌合家族的传播。","authors":"Wu Zubo, Jie Liu, Yi Liu, Xiaoli Wang, Defeng Shu","doi":"10.1089/dna.2024.0280","DOIUrl":null,"url":null,"abstract":"<p><p>This study presents a case of autosomal dominant polycystic kidney disease (ADPKD) involving a mosaic microdeletion in the <i>PKD1</i> gene and explores the application of long-read sequencing technologies for haplotype construction and preimplantation genetic testing (PGT). We report on a family where the proband was clinically diagnosed with PKD and found to have a partial deletion of the <i>PKD1</i> gene because of the mosaic deletion mutation of <i>PKD1</i> in the mother of the proband. Utilizing Oxford Nanopore long-read sequencing, we successfully constructed the haplotype of the deleted fragment region and identified an unaffected embryo for transplantation, resulting in a successful pregnancy. The prenatal genetic diagnosis confirmed the absence of deletion abnormalities in the fetus. Our findings underscore the significance of integrating advanced genomic technologies into clinical practice for PGT in ADPKD, particularly in cases involving partial deletion of X chromosome mosaic embryo transferred or complex structural variants. This approach not only prevents the transmission of ADPKD but also demonstrates the utility of long-read sequencing in overcoming the limitations of traditional PGT methods. Further research is warranted to evaluate the broader application of long-read sequencing for other monogenic disorders and to refine these techniques for enhanced diagnostic precision and clinical outcomes.</p>","PeriodicalId":93981,"journal":{"name":"DNA and cell biology","volume":" ","pages":"238-248"},"PeriodicalIF":2.6000,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Utilizing Long-Read Sequencing for Haplotype Construction and Prevention of Autosomal Dominant Polycystic Kidney Disease Transmission in Mosaicism Family.\",\"authors\":\"Wu Zubo, Jie Liu, Yi Liu, Xiaoli Wang, Defeng Shu\",\"doi\":\"10.1089/dna.2024.0280\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>This study presents a case of autosomal dominant polycystic kidney disease (ADPKD) involving a mosaic microdeletion in the <i>PKD1</i> gene and explores the application of long-read sequencing technologies for haplotype construction and preimplantation genetic testing (PGT). We report on a family where the proband was clinically diagnosed with PKD and found to have a partial deletion of the <i>PKD1</i> gene because of the mosaic deletion mutation of <i>PKD1</i> in the mother of the proband. Utilizing Oxford Nanopore long-read sequencing, we successfully constructed the haplotype of the deleted fragment region and identified an unaffected embryo for transplantation, resulting in a successful pregnancy. The prenatal genetic diagnosis confirmed the absence of deletion abnormalities in the fetus. Our findings underscore the significance of integrating advanced genomic technologies into clinical practice for PGT in ADPKD, particularly in cases involving partial deletion of X chromosome mosaic embryo transferred or complex structural variants. This approach not only prevents the transmission of ADPKD but also demonstrates the utility of long-read sequencing in overcoming the limitations of traditional PGT methods. Further research is warranted to evaluate the broader application of long-read sequencing for other monogenic disorders and to refine these techniques for enhanced diagnostic precision and clinical outcomes.</p>\",\"PeriodicalId\":93981,\"journal\":{\"name\":\"DNA and cell biology\",\"volume\":\" \",\"pages\":\"238-248\"},\"PeriodicalIF\":2.6000,\"publicationDate\":\"2025-05-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"DNA and cell biology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1089/dna.2024.0280\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/4/2 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"DNA and cell biology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1089/dna.2024.0280","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/4/2 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

摘要

本研究报道了一例常染色体显性多囊肾病(ADPKD),涉及PKD1基因的镶嵌微缺失,并探讨了长读测序技术在单倍型构建和植入前基因检测(PGT)中的应用。我们报告了一个先证者被临床诊断为PKD的家庭,并发现由于先证者母亲PKD1的马赛克缺失突变,PKD1基因部分缺失。利用Oxford Nanopore长读测序技术,我们成功构建了缺失片段区域的单倍型,并鉴定出未受影响的胚胎进行移植,成功怀孕。产前遗传诊断证实胎儿没有缺失异常。我们的研究结果强调了将先进的基因组技术整合到ADPKD的PGT临床实践中的重要性,特别是在涉及X染色体嵌合体胚胎移植部分缺失或复杂结构变异的情况下。这种方法不仅可以防止ADPKD的传播,而且还证明了长读测序在克服传统PGT方法的局限性方面的实用性。需要进一步的研究来评估长读测序在其他单基因疾病中的广泛应用,并改进这些技术以提高诊断精度和临床结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Utilizing Long-Read Sequencing for Haplotype Construction and Prevention of Autosomal Dominant Polycystic Kidney Disease Transmission in Mosaicism Family.

This study presents a case of autosomal dominant polycystic kidney disease (ADPKD) involving a mosaic microdeletion in the PKD1 gene and explores the application of long-read sequencing technologies for haplotype construction and preimplantation genetic testing (PGT). We report on a family where the proband was clinically diagnosed with PKD and found to have a partial deletion of the PKD1 gene because of the mosaic deletion mutation of PKD1 in the mother of the proband. Utilizing Oxford Nanopore long-read sequencing, we successfully constructed the haplotype of the deleted fragment region and identified an unaffected embryo for transplantation, resulting in a successful pregnancy. The prenatal genetic diagnosis confirmed the absence of deletion abnormalities in the fetus. Our findings underscore the significance of integrating advanced genomic technologies into clinical practice for PGT in ADPKD, particularly in cases involving partial deletion of X chromosome mosaic embryo transferred or complex structural variants. This approach not only prevents the transmission of ADPKD but also demonstrates the utility of long-read sequencing in overcoming the limitations of traditional PGT methods. Further research is warranted to evaluate the broader application of long-read sequencing for other monogenic disorders and to refine these techniques for enhanced diagnostic precision and clinical outcomes.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信