Hiroki Ura , Sumihito Togi , Hisayo Hatanaka , Yo Niida
{"title":"从一名携带 KAT6B 基因杂合框移位突变的 Say-Barber-Biesecker-Young-Simpson 奥多综合征变异型患者体内建立人类诱导多能干细胞系 KMUGMCi008-A","authors":"Hiroki Ura , Sumihito Togi , Hisayo Hatanaka , Yo Niida","doi":"10.1016/j.scr.2025.103671","DOIUrl":null,"url":null,"abstract":"<div><div>Ohdo syndrome Say-Barver-Biesecker-Young-Simpson (SBBYS) variant is a rare autosomal dominant disorder characterized mainly by intellectual disability, developmental delays, contractures of the knees and hips contractures, thyroid dysfunction, and dysmorphic appearance. The Ohdo syndrome SBBYS variant is caused by heterozygous loss of function mutation in the <em>KAT6B</em> gene. The peripheral blood mononuclear cells from a patient carrying heterozygous frameshift mutation of the <em>KAT6B</em> gene were reprogrammed using the CytoTune-iPS2.0 Sendai Reprogramming Kit. The mutation in the <em>KAT6B gene</em> causes the abnormal protein variant. The established human induced pluripotent cell line allow proper <em>in vitro</em> disease modelling of Ohdo syndrome SBBYS variant.</div></div>","PeriodicalId":21843,"journal":{"name":"Stem cell research","volume":"84 ","pages":"Article 103671"},"PeriodicalIF":0.8000,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Establishment of a human induced pluripotent stem cell line, KMUGMCi008-A, from a patient with A Say-Barber-Biesecker-Young-Simpson variant of Ohdo syndrome bearing heterozygous frameshift mutation in the KAT6B gene\",\"authors\":\"Hiroki Ura , Sumihito Togi , Hisayo Hatanaka , Yo Niida\",\"doi\":\"10.1016/j.scr.2025.103671\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Ohdo syndrome Say-Barver-Biesecker-Young-Simpson (SBBYS) variant is a rare autosomal dominant disorder characterized mainly by intellectual disability, developmental delays, contractures of the knees and hips contractures, thyroid dysfunction, and dysmorphic appearance. The Ohdo syndrome SBBYS variant is caused by heterozygous loss of function mutation in the <em>KAT6B</em> gene. The peripheral blood mononuclear cells from a patient carrying heterozygous frameshift mutation of the <em>KAT6B</em> gene were reprogrammed using the CytoTune-iPS2.0 Sendai Reprogramming Kit. The mutation in the <em>KAT6B gene</em> causes the abnormal protein variant. The established human induced pluripotent cell line allow proper <em>in vitro</em> disease modelling of Ohdo syndrome SBBYS variant.</div></div>\",\"PeriodicalId\":21843,\"journal\":{\"name\":\"Stem cell research\",\"volume\":\"84 \",\"pages\":\"Article 103671\"},\"PeriodicalIF\":0.8000,\"publicationDate\":\"2025-02-04\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Stem cell research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1873506125000212\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem cell research","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1873506125000212","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
Ohdo综合征sayer - barver - biesecker - young - simpson (SBBYS)变异体是一种罕见的常染色体显性遗传病,主要表现为智力障碍、发育迟缓、膝和髋挛缩、甲状腺功能障碍和外观畸形。Ohdo综合征SBBYS变异是由KAT6B基因杂合性功能缺失突变引起的。使用CytoTune-iPS2.0仙台重编程试剂盒对携带KAT6B基因杂合移码突变的患者外周血单个核细胞进行重编程。KAT6B基因的突变导致了异常的蛋白质变异。建立的人类诱导多能细胞系允许适当的Ohdo综合征SBBYS变异的体外疾病建模。
Establishment of a human induced pluripotent stem cell line, KMUGMCi008-A, from a patient with A Say-Barber-Biesecker-Young-Simpson variant of Ohdo syndrome bearing heterozygous frameshift mutation in the KAT6B gene
Ohdo syndrome Say-Barver-Biesecker-Young-Simpson (SBBYS) variant is a rare autosomal dominant disorder characterized mainly by intellectual disability, developmental delays, contractures of the knees and hips contractures, thyroid dysfunction, and dysmorphic appearance. The Ohdo syndrome SBBYS variant is caused by heterozygous loss of function mutation in the KAT6B gene. The peripheral blood mononuclear cells from a patient carrying heterozygous frameshift mutation of the KAT6B gene were reprogrammed using the CytoTune-iPS2.0 Sendai Reprogramming Kit. The mutation in the KAT6B gene causes the abnormal protein variant. The established human induced pluripotent cell line allow proper in vitro disease modelling of Ohdo syndrome SBBYS variant.
期刊介绍:
Stem Cell Research is dedicated to publishing high-quality manuscripts focusing on the biology and applications of stem cell research. Submissions to Stem Cell Research, may cover all aspects of stem cells, including embryonic stem cells, tissue-specific stem cells, cancer stem cells, developmental studies, stem cell genomes, and translational research. Stem Cell Research publishes 6 issues a year.