通过外显子组测序鉴定科威特近亲家庭唇腭裂患者新颖和罕见的基因变异。

IF 1.7 4区 生物学 Q3 GENETICS & HEREDITY
Lateefa Alkharafi, Suzanne Al-Bustan, Sumaya Alkanderi, Reem M Elshafie, Heba Dannoun, Hadil Alrohaif, Alaa-Eldin Elshafey, Amal Alwadani, Saud Alhasawi, Nour Aljeraiwi, Khaled Alotaibi, Hala Hamdan, Dedeepya Vaka, Ugur Hodoglugil, Dario Boffelli, Kimberly K Diaz Perez, Elizabeth J Leslie-Clarkson, Ophir D Klein
{"title":"通过外显子组测序鉴定科威特近亲家庭唇腭裂患者新颖和罕见的基因变异。","authors":"Lateefa Alkharafi, Suzanne Al-Bustan, Sumaya Alkanderi, Reem M Elshafie, Heba Dannoun, Hadil Alrohaif, Alaa-Eldin Elshafey, Amal Alwadani, Saud Alhasawi, Nour Aljeraiwi, Khaled Alotaibi, Hala Hamdan, Dedeepya Vaka, Ugur Hodoglugil, Dario Boffelli, Kimberly K Diaz Perez, Elizabeth J Leslie-Clarkson, Ophir D Klein","doi":"10.1002/ajmg.a.64268","DOIUrl":null,"url":null,"abstract":"<p><p>The prevalence of Cleft Lip with or without Cleft Palate (CLP) varies by geography and ethnicity, particularly in consanguineous populations. This study aimed to identify gene variants contributing to the development of both syndromic (SCLP) and non-syndromic (NSCLP) forms of the condition in 20 consanguineous Kuwaiti families. We used exome sequencing and assessed the variants' predicted functional roles. Seven rare gene variants were identified across all cases, including one novel variant (LRRC32) in a syndromic case and three variants (SH3PXD2A, DUOX1, MSX2) in non-syndromic cases. These genes are predicted to be involved in cellular metabolism, differentiation, signaling, and regulatory mechanisms. Four genes (SH3PXD2A, DUOX1, MSX2, and ACACB) identified in the non-syndromic cases were found to have a significant association with co-expression of the transcription factor TP63. These findings provide a foundation for further investigation into the functional roles of these novel variants in the development of NSCLP, particularly DUOX1, which has not been previously reported in CLP.</p>","PeriodicalId":7507,"journal":{"name":"American Journal of Medical Genetics Part A","volume":" ","pages":"e64268"},"PeriodicalIF":1.7000,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Identification of Novel and Rare Gene Variants in Cleft Lip/Palate Patients From Kuwaiti Consanguineous Families by Exome Sequencing.\",\"authors\":\"Lateefa Alkharafi, Suzanne Al-Bustan, Sumaya Alkanderi, Reem M Elshafie, Heba Dannoun, Hadil Alrohaif, Alaa-Eldin Elshafey, Amal Alwadani, Saud Alhasawi, Nour Aljeraiwi, Khaled Alotaibi, Hala Hamdan, Dedeepya Vaka, Ugur Hodoglugil, Dario Boffelli, Kimberly K Diaz Perez, Elizabeth J Leslie-Clarkson, Ophir D Klein\",\"doi\":\"10.1002/ajmg.a.64268\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>The prevalence of Cleft Lip with or without Cleft Palate (CLP) varies by geography and ethnicity, particularly in consanguineous populations. This study aimed to identify gene variants contributing to the development of both syndromic (SCLP) and non-syndromic (NSCLP) forms of the condition in 20 consanguineous Kuwaiti families. We used exome sequencing and assessed the variants' predicted functional roles. Seven rare gene variants were identified across all cases, including one novel variant (LRRC32) in a syndromic case and three variants (SH3PXD2A, DUOX1, MSX2) in non-syndromic cases. These genes are predicted to be involved in cellular metabolism, differentiation, signaling, and regulatory mechanisms. Four genes (SH3PXD2A, DUOX1, MSX2, and ACACB) identified in the non-syndromic cases were found to have a significant association with co-expression of the transcription factor TP63. These findings provide a foundation for further investigation into the functional roles of these novel variants in the development of NSCLP, particularly DUOX1, which has not been previously reported in CLP.</p>\",\"PeriodicalId\":7507,\"journal\":{\"name\":\"American Journal of Medical Genetics Part A\",\"volume\":\" \",\"pages\":\"e64268\"},\"PeriodicalIF\":1.7000,\"publicationDate\":\"2025-10-03\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"American Journal of Medical Genetics Part A\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://doi.org/10.1002/ajmg.a.64268\",\"RegionNum\":4,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"American Journal of Medical Genetics Part A","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1002/ajmg.a.64268","RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0

摘要

唇裂伴或不伴腭裂(CLP)的患病率因地理和种族而异,特别是在近亲人群中。本研究旨在确定在20个科威特近亲家庭中导致综合征(SCLP)和非综合征(NSCLP)形式的基因变异。我们使用外显子组测序并评估变异的预测功能作用。在所有病例中鉴定出7种罕见基因变异,包括综合征病例中的1种新变异(LRRC32)和非综合征病例中的3种变异(SH3PXD2A、DUOX1、MSX2)。这些基因被预测参与细胞代谢、分化、信号传导和调节机制。在非综合征病例中发现的四个基因(SH3PXD2A、DUOX1、MSX2和ACACB)与转录因子TP63的共表达有显著相关性。这些发现为进一步研究这些新变异在NSCLP发展中的功能作用提供了基础,特别是DUOX1,这是以前未在CLP中报道的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Identification of Novel and Rare Gene Variants in Cleft Lip/Palate Patients From Kuwaiti Consanguineous Families by Exome Sequencing.

The prevalence of Cleft Lip with or without Cleft Palate (CLP) varies by geography and ethnicity, particularly in consanguineous populations. This study aimed to identify gene variants contributing to the development of both syndromic (SCLP) and non-syndromic (NSCLP) forms of the condition in 20 consanguineous Kuwaiti families. We used exome sequencing and assessed the variants' predicted functional roles. Seven rare gene variants were identified across all cases, including one novel variant (LRRC32) in a syndromic case and three variants (SH3PXD2A, DUOX1, MSX2) in non-syndromic cases. These genes are predicted to be involved in cellular metabolism, differentiation, signaling, and regulatory mechanisms. Four genes (SH3PXD2A, DUOX1, MSX2, and ACACB) identified in the non-syndromic cases were found to have a significant association with co-expression of the transcription factor TP63. These findings provide a foundation for further investigation into the functional roles of these novel variants in the development of NSCLP, particularly DUOX1, which has not been previously reported in CLP.

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
CiteScore
3.50
自引率
5.00%
发文量
432
审稿时长
2-4 weeks
期刊介绍: The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts: Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders. Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .
×
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