• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

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

CYP11A1 缺陷导致的男性患儿出现原发性肾上腺皮质功能减退伴正常男性外生殖器。

Primary adrenal insufficiency with normal male external genitalia in a boy with CYP11A1 deficiency.

机构信息

Department of Paediatric Endocrinology, Valluvanad Hospital Complex Limited, Palakkad, Kerala, India.

West Midlands Deanery, Edgbaston, Birmingham, UK.

出版信息

BMJ Case Rep. 2024 Sep 19;17(9):e261512. doi: 10.1136/bcr-2024-261512.

DOI:10.1136/bcr-2024-261512
PMID:39304217
Abstract

The first and rate-limiting step of steroid hormone biosynthesis is catalysed by mitochondrial cytochrome P450 side-chain cleavage enzyme (CYP11A1). CYP11A1 deficiency is commonly associated with adrenal insufficiency and, in 46 XY individuals, with variable degrees of differences in sex development (DSD). Here, we present a case report of a preadolescent male who presented to our emergency outpatient department in a state of decompensated shock necessitating ionotropic support. Further evaluation confirmed primary adrenal insufficiency. Subsequent clinical exome sequencing uncovered a compound heterozygous mutation in exons one and five of the CYP11A1 gene. This case highlights the varied presentation of CYP11A1 deficiency, showing that it can present as adrenal insufficiency without DSD.

摘要

甾体激素生物合成的第一步和限速步骤是由线粒体细胞色素 P450 侧链裂解酶(CYP11A1)催化的。CYP11A1 缺乏通常与肾上腺功能不全有关,在 46 XY 个体中,与性别发育(DSD)的不同程度差异有关。在这里,我们报告了一例青春期前男性的病例,他因失代偿性休克到我们的急诊门诊就诊,需要离子支持。进一步的评估证实了原发性肾上腺功能不全。随后的临床外显子组测序发现 CYP11A1 基因的第一和第五外显子存在复合杂合突变。这个病例突出了 CYP11A1 缺乏的不同表现,表明它可以表现为没有 DSD 的肾上腺功能不全。

相似文献

1
Primary adrenal insufficiency with normal male external genitalia in a boy with CYP11A1 deficiency.CYP11A1 缺陷导致的男性患儿出现原发性肾上腺皮质功能减退伴正常男性外生殖器。
BMJ Case Rep. 2024 Sep 19;17(9):e261512. doi: 10.1136/bcr-2024-261512.
2
Normal male external genitalia do not rule out CYP11A1 deficiency.正常男性外生殖器不能排除CYP11A1缺乏症。
BMJ Case Rep. 2019 Jul 8;12(7):e228235. doi: 10.1136/bcr-2018-228235.
3
A novel entity of clinically isolated adrenal insufficiency caused by a partially inactivating mutation of the gene encoding for P450 side chain cleavage enzyme (CYP11A1).一种新的临床孤立性肾上腺功能不全的实体,由编码 P450 侧链裂解酶(CYP11A1)的基因部分失活突变引起。
J Clin Endocrinol Metab. 2011 Nov;96(11):E1798-806. doi: 10.1210/jc.2011-1277. Epub 2011 Aug 31.
4
Delayed diagnosis of adrenal insufficiency in a patient with severe penoscrotal hypospadias due to two novel P450 side-change cleavage enzyme (CYP11A1) mutations (p.R360W; p.R405X).由于两个新的 P450 侧链裂解酶(CYP11A1)突变(p.R360W;p.R405X),导致严重的阴茎阴囊型尿道下裂患者肾上腺皮质功能不全的诊断延迟。
Eur J Endocrinol. 2012 Dec;167(6):881-5. doi: 10.1530/EJE-12-0450. Epub 2012 Sep 11.
5
Heterozygous mutations in the cholesterol side-chain cleavage enzyme gene (CYP11A1) can cause transient adrenal insufficiency and life-threatening failure to thrive.胆固醇侧链裂解酶基因(CYP11A1)杂合突变可导致短暂性肾上腺皮质功能不全和危及生命的生长发育迟缓。
Hormones (Athens). 2018 Sep;17(3):419-421. doi: 10.1007/s42000-018-0048-y. Epub 2018 Jul 11.
6
A novel homozygous mutation in CYP11A1 gene is associated with late-onset adrenal insufficiency and hypospadias in a 46,XY patient.一名46,XY男性患者中,CYP11A1基因的一种新型纯合突变与迟发性肾上腺皮质功能不全和尿道下裂相关。
J Clin Endocrinol Metab. 2009 Mar;94(3):936-9. doi: 10.1210/jc.2008-1118. Epub 2008 Dec 30.
7
Adrenal Insufficiency, Sex Reversal, and Angelman Syndrome due to Uniparental Disomy Unmasking a Mutation in CYP11A1.由于 CYP11A1 基因突变导致的单亲二体性、肾上腺皮质功能不全、性反转和 Angelman 综合征。
Horm Res Paediatr. 2018;89(3):205-210. doi: 10.1159/000487638. Epub 2018 Mar 22.
8
Can Digenic, Tri-Allelic Inheritance of Variants in and Give Rise to Primary Adrenal Insufficiency? A Case Report.是否可以通过 和 基因的双基因、三等位基因遗传变异导致原发性肾上腺功能不全?病例报告。
Front Endocrinol (Lausanne). 2022 Mar 28;13:860055. doi: 10.3389/fendo.2022.860055. eCollection 2022.
9
Homozygous mutation of P450 side-chain cleavage enzyme gene (CYP11A1) in 46, XY patient with adrenal insufficiency, complete sex reversal, and agenesis of corpus callosum.一名46,XY的患者,患有肾上腺功能不全、完全性性别反转和胼胝体发育不全,其细胞色素P450侧链裂解酶基因(CYP11A1)存在纯合突变。
J Clin Endocrinol Metab. 2006 Aug;91(8):2821-6. doi: 10.1210/jc.2005-2230. Epub 2006 May 16.
10
A novel splice site variant in CYP11A1 in trans with the p.E314K variant in a male patient with congenital adrenal insufficiency.一名先天性肾上腺皮质功能减退男性患者中,CYP11A1基因存在一种与p.E314K变异呈反式关系的新型剪接位点变异。
Mol Genet Genomic Med. 2017 Nov;5(6):781-787. doi: 10.1002/mgg3.322. Epub 2017 Jul 20.