{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T23:29:51Z","timestamp":1786663791244,"version":"build-2736575974"},"reference-count":46,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T00:00:00Z","timestamp":1762387200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000038","name":"FDA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000038","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["gimjournal.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Genetics in Medicine"],"published-print":{"date-parts":[[2026,1]]},"DOI":"10.1016\/j.gim.2025.101623","type":"journal-article","created":{"date-parts":[[2025,10,25]],"date-time":"2025-10-25T01:37:31Z","timestamp":1761356251000},"page":"101623","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":1,"title":["De novo rare EMX2 variants lead to idiopathic hypogonadotropic hypogonadism"],"prefix":"10.1016","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5992-2120","authenticated-orcid":false,"given":"Maria","family":"Stamou","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miranda","family":"Tompkins","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hannah","family":"Bow","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Kearney","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maleeha","family":"Akram","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harrison","family":"Brand","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuefang","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shadi","family":"Zaheri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neoklis A.","family":"Georgopoulos","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Odelia","family":"Chorin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yulia","family":"Khavkin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tal","family":"Kedar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret F.","family":"Lippincott","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lacey","family":"Plummer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Talkowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yiping","family":"Shen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doris K.","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ravikumar","family":"Balasubramanian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Wray","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie B.","family":"Seminara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"5","key":"10.1016\/j.gim.2025.101623_bib1","doi-asserted-by":"crossref","DOI":"10.1111\/jne.13080","article-title":"Review of human genetic and clinical studies directly relevant to GnRH signalling","volume":"34","author":"Seminara","year":"2022","journal-title":"J Neuroendocrinol"},{"issue":"8","key":"10.1016\/j.gim.2025.101623_bib2","doi-asserted-by":"crossref","first-page":"3479","DOI":"10.1210\/en.2010-0022","article-title":"Minireview: kisspeptin\/neurokinin B\/dynorphin (KNDy) cells of the arcuate nucleus: a central node in the control of gonadotropin-releasing hormone secretion","volume":"151","author":"Lehman","year":"2010","journal-title":"Endocrinology"},{"issue":"1","key":"10.1016\/j.gim.2025.101623_bib3","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1080\/17446651.2022.2023008","article-title":"Current concepts surrounding neonatal hormone therapy for boys with congenital hypogonadotropic hypogonadism","volume":"17","author":"Swee","year":"2022","journal-title":"Expert Rev Endocrinol Metab"},{"issue":"5","key":"10.1016\/j.gim.2025.101623_bib4","doi-asserted-by":"crossref","first-page":"E943","DOI":"10.1210\/jc.2012-4116","article-title":"Prioritizing genetic testing in patients with Kallmann syndrome using clinical phenotypes","volume":"98","author":"Costa-Barbosa","year":"2013","journal-title":"J Clin Endocrinol Metab"},{"issue":"8","key":"10.1016\/j.gim.2025.101623_bib5","doi-asserted-by":"crossref","first-page":"2228","DOI":"10.1210\/clinem\/dgac300","article-title":"Prevalence and phenotypic effects of copy number variants in isolated hypogonadotropic hypogonadism","volume":"107","author":"Stamou","year":"2022","journal-title":"J Clin Endocrinol Metab"},{"issue":"4","key":"10.1016\/j.gim.2025.101623_bib6","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1038\/ng1122","article-title":"Loss-of-function mutations in FGFR1 cause autosomal dominant Kallmann syndrome","volume":"33","author":"Dod\u00e9","year":"2003","journal-title":"Nat Genet"},{"issue":"17","key":"10.1016\/j.gim.2025.101623_bib7","doi-asserted-by":"crossref","first-page":"1614","DOI":"10.1056\/NEJMoa035322","article-title":"The GPR54 gene as a regulator of puberty","volume":"349","author":"Seminara","year":"2003","journal-title":"N Engl J Med"},{"issue":"3","key":"10.1016\/j.gim.2025.101623_bib8","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1038\/ng.306","article-title":"TAC3 and TACR3 mutations in familial hypogonadotropic hypogonadism reveal a key role for neurokinin B in the central control of reproduction","volume":"41","author":"Topaloglu","year":"2009","journal-title":"Nat Genet"},{"issue":"6344","key":"10.1016\/j.gim.2025.101623_bib9","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1038\/353529a0","article-title":"A gene deleted in Kallmann\u2019s syndrome shares homology with neural cell adhesion and axonal path-finding molecules","volume":"353","author":"Franco","year":"1991","journal-title":"Nature"},{"issue":"7809","key":"10.1016\/j.gim.2025.101623_bib10","doi-asserted-by":"crossref","first-page":"434","DOI":"10.1038\/s41586-020-2308-7","article-title":"The mutational constraint spectrum quantified from variation in 141,456 humans","volume":"581","author":"Karczewski","year":"2020","journal-title":"Nature"},{"issue":"1-2","key":"10.1016\/j.gim.2025.101623_bib11","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.mce.2011.07.012","article-title":"Isolated GnRH deficiency: a disease model serving as a unique prism into the systems biology of the GnRH neuronal network","volume":"346","author":"Balasubramanian","year":"2011","journal-title":"Mol Cell Endocrinol"},{"issue":"6","key":"10.1016\/j.gim.2025.101623_bib12","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1016\/j.ajhg.2015.04.013","article-title":"Individualized iterative phenotyping for genome-wide analysis of loss-of-function mutations","volume":"96","author":"Johnston","year":"2015","journal-title":"Am J Hum Genet"},{"issue":"6","key":"10.1016\/j.gim.2025.101623_bib13","first-page":"698","article-title":"seqr: a web-based analysis and collaboration tool for rare disease genomics","volume":"43","author":"Pais","year":"2022","journal-title":"Hum Mutat"},{"issue":"4","key":"10.1016\/j.gim.2025.101623_bib14","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1016\/j.ajhg.2021.02.012","article-title":"De novo structural mutation rates and gamete-of-origin biases revealed through genome sequencing of 2,396 families","volume":"108","author":"Belyeu","year":"2021","journal-title":"Am J Hum Genet"},{"issue":"3","key":"10.1016\/j.gim.2025.101623_bib15","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1086\/519795","article-title":"PLINK: a tool set for whole-genome association and population-based linkage analyses","volume":"81","author":"Purcell","year":"2007","journal-title":"Am J Hum Genet"},{"issue":"D1","key":"10.1016\/j.gim.2025.101623_bib16","doi-asserted-by":"crossref","first-page":"D998","DOI":"10.1093\/nar\/gkae1070","article-title":"The NHGRI-EBI GWAS catalog: standards for reusability, sustainability and diversity","volume":"53","author":"Cerezo","year":"2025","journal-title":"Nucleic Acids Res"},{"issue":"D1","key":"10.1016\/j.gim.2025.101623_bib17","doi-asserted-by":"crossref","first-page":"D1005","DOI":"10.1093\/nar\/gky1120","article-title":"The NHGRI-EBI GWAS catalog of published genome-wide association studies, targeted arrays and summary statistics 2019","volume":"47","author":"Buniello","year":"2019","journal-title":"Nucleic Acids Res"},{"issue":"Database issue","key":"10.1016\/j.gim.2025.101623_bib18","doi-asserted-by":"crossref","first-page":"D514","DOI":"10.1093\/nar\/gki033","article-title":"Online Mendelian Inheritance in Man (OMIM), a knowledgebase of human genes and genetic disorders","volume":"33","author":"Hamosh","year":"2005","journal-title":"Nucleic Acids Res"},{"issue":"Database issue","key":"10.1016\/j.gim.2025.101623_bib19","doi-asserted-by":"crossref","first-page":"D980","DOI":"10.1093\/nar\/gkt1113","article-title":"ClinVar: public archive of relationships among sequence variation and human phenotype","volume":"42","author":"Landrum","year":"2014","journal-title":"Nucleic Acids Res"},{"issue":"D1","key":"10.1016\/j.gim.2025.101623_bib20","doi-asserted-by":"crossref","first-page":"D1353","DOI":"10.1093\/nar\/gkac1046","article-title":"The next-generation Open Targets Platform: reimagined, redesigned, rebuilt","volume":"51","author":"Ochoa","year":"2023","journal-title":"Nucleic Acids Res"},{"issue":"23","key":"10.1016\/j.gim.2025.101623_bib21","doi-asserted-by":"crossref","first-page":"2235","DOI":"10.1056\/NEJMsr1406261","article-title":"ClinGen--the clinical genome resource","volume":"372","author":"Rehm","year":"2015","journal-title":"N Engl J Med"},{"issue":"20","key":"10.1016\/j.gim.2025.101623_bib22","doi-asserted-by":"crossref","first-page":"3207","DOI":"10.1093\/bioinformatics\/btw373","article-title":"PhenoScanner: a database of human genotype-phenotype associations","volume":"32","author":"Staley","year":"2016","journal-title":"Bioinformatics"},{"issue":"10","key":"10.1016\/j.gim.2025.101623_bib23","doi-asserted-by":"crossref","first-page":"928","DOI":"10.1002\/humu.22844","article-title":"GeneMatcher: a matching tool for connecting investigators with an interest in the same gene","volume":"36","author":"Sobreira","year":"2015","journal-title":"Hum Mutat"},{"issue":"3","key":"10.1016\/j.gim.2025.101623_bib24","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2009493118","article-title":"Drebrin regulates cytoskeleton dynamics in migrating neurons through interaction with CXCR4","volume":"118","author":"Shan","year":"2021","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"24","key":"10.1016\/j.gim.2025.101623_bib25","doi-asserted-by":"crossref","first-page":"4759","DOI":"10.1093\/hmg\/ddr403","article-title":"Dysregulation of Semaphorin 7A\/\u03b21-integrin signaling leads to defective GnRH-1 cell migration, abnormal gonadal development and altered fertility","volume":"20","author":"Messina","year":"2011","journal-title":"Hum Mol Genet"},{"issue":"14","key":"10.1016\/j.gim.2025.101623_bib26","doi-asserted-by":"crossref","first-page":"1824","DOI":"10.1101\/gad.14.14.1824","article-title":"Novel gene expressed in nasal region influences outgrowth of olfactory axons and migration of luteinizing hormone-releasing hormone (LHRH) neurons","volume":"14","author":"Kramer","year":"2000","journal-title":"Genes Dev"},{"issue":"5","key":"10.1016\/j.gim.2025.101623_bib27","doi-asserted-by":"crossref","first-page":"1956","DOI":"10.1210\/en.2015-1846","article-title":"CCDC141 mutation identified in anosmic hypogonadotropic hypogonadism (Kallmann syndrome) alters GnRH neuronal migration","volume":"157","author":"Hutchins","year":"2016","journal-title":"Endocrinology"},{"issue":"21","key":"10.1016\/j.gim.2025.101623_bib28","doi-asserted-by":"crossref","first-page":"5097","DOI":"10.1523\/JNEUROSCI.0239-05.2005","article-title":"Emx2 and Pax6 function in cooperation with Otx2 and Otx1 to develop caudal forebrain primordium that includes future archipallium","volume":"25","author":"Kimura","year":"2005","journal-title":"J Neurosci"},{"issue":"12","key":"10.1016\/j.gim.2025.101623_bib29","doi-asserted-by":"crossref","first-page":"3893","DOI":"10.1242\/dev.122.12.3893","article-title":"Dentate gyrus formation requires Emx2","volume":"122","author":"Pellegrini","year":"1996","journal-title":"Development"},{"issue":"2","key":"10.1016\/j.gim.2025.101623_bib30","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1523\/JNEUROSCI.4698-10.2011","article-title":"A role for FE65 in controlling GnRH-1 neurogenesis","volume":"31","author":"Forni","year":"2011","journal-title":"J Neurosci"},{"issue":"5","key":"10.1016\/j.gim.2025.101623_bib31","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1038\/gim.2015.30","article-title":"Standards and guidelines for the interpretation of sequence variants: a joint consensus recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology","volume":"17","author":"Richards","year":"2015","journal-title":"Genet Med"},{"issue":"3","key":"10.1016\/j.gim.2025.101623_bib32","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.164324","article-title":"Heterozygous mutations in SOX2 may cause idiopathic hypogonadotropic hypogonadism via dominant-negative mechanisms","volume":"8","author":"Cassin","year":"2023","journal-title":"JCI Insight"},{"issue":"20","key":"10.1016\/j.gim.2025.101623_bib33","doi-asserted-by":"crossref","first-page":"8132","DOI":"10.1073\/pnas.86.20.8132","article-title":"Evidence that cells expressing luteinizing hormone-releasing hormone mRNA in the mouse are derived from progenitor cells in the olfactory placode","volume":"86","author":"Wray","year":"1989","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"7","key":"10.1016\/j.gim.2025.101623_bib34","doi-asserted-by":"crossref","first-page":"2541","DOI":"10.1002\/j.1460-2075.1992.tb05319.x","article-title":"Two vertebrate homeobox genes related to the Drosophila empty spiracles gene are expressed in the embryonic cerebral cortex","volume":"11","author":"Simeone","year":"1992","journal-title":"EMBO J"},{"issue":"2","key":"10.1016\/j.gim.2025.101623_bib35","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1016\/S0925-4773(98)00141-5","article-title":"EMX2 protein in the developing mouse brain and olfactory area","volume":"77","author":"Mallamaci","year":"1998","journal-title":"Mech Dev"},{"issue":"5","key":"10.1016\/j.gim.2025.101623_bib36","doi-asserted-by":"crossref","DOI":"10.1111\/jne.13087","article-title":"Development of the gonadotropin-releasing hormone system","volume":"34","author":"Duittoz","year":"2022","journal-title":"J Neuroendocrinol"},{"issue":"14","key":"10.1016\/j.gim.2025.101623_bib37","doi-asserted-by":"crossref","first-page":"6461","DOI":"10.1093\/nar\/gks295","article-title":"Emx2 is a dose-dependent negative regulator of Sox2 telencephalic enhancers","volume":"40","author":"Mariani","year":"2012","journal-title":"Nucleic Acids Res"},{"issue":"19","key":"10.1016\/j.gim.2025.101623_bib38","doi-asserted-by":"crossref","first-page":"19156","DOI":"10.1074\/jbc.M502004200","article-title":"Developmental regulation of gonadotropin-releasing hormone gene expression by the MSX and DLX homeodomain protein families","volume":"280","author":"Givens","year":"2005","journal-title":"J Biol Chem"},{"issue":"4","key":"10.1016\/j.gim.2025.101623_bib39","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1002\/cne.10746","article-title":"Prenatal development of the rodent rostral migratory stream","volume":"463","author":"Pencea","year":"2003","journal-title":"J Comp Neurol"},{"key":"10.1016\/j.gim.2025.101623_bib40","doi-asserted-by":"crossref","DOI":"10.3389\/fendo.2023.1203542","article-title":"POU6F2 mutation in humans with pubertal failure alters GnRH transcript expression","volume":"14","author":"Cho","year":"2023","journal-title":"Front Endocrinol (Lausanne)"},{"issue":"3","key":"10.1016\/j.gim.2025.101623_bib41","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1016\/j.fertnstert.2014.11.030","article-title":"Nonsense mutation of EMX2 is potential causative for uterus didelphysis: first molecular explanation for isolated incomplete m\u00fcllerian fusion","volume":"103","author":"Liu","year":"2015","journal-title":"Fertil Steril"},{"issue":"1-3","key":"10.1016\/j.gim.2025.101623_bib42","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1006\/geno.2001.6590","article-title":"Characterization of the homeodomain gene EMX2: sequence conservation, expression analysis, and a search for mutations in endometrial cancers","volume":"76","author":"Noonan","year":"2001","journal-title":"Genomics"},{"issue":"9","key":"10.1016\/j.gim.2025.101623_bib43","doi-asserted-by":"crossref","first-page":"1653","DOI":"10.1242\/dev.124.9.1653","article-title":"Defects of urogenital development in mice lacking Emx2","volume":"124","author":"Miyamoto","year":"1997","journal-title":"Development"},{"issue":"12","key":"10.1016\/j.gim.2025.101623_bib44","doi-asserted-by":"crossref","first-page":"1313","DOI":"10.1002\/ajmg.a.31767","article-title":"Comprehensive EMX2 genotyping of a large schizencephaly case series","volume":"143A","author":"Tietjen","year":"2007","journal-title":"Am J Med Genet A"},{"issue":"7601","key":"10.1016\/j.gim.2025.101623_bib45","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1038\/nature17939","article-title":"Parkinson-associated risk variant in distal enhancer of \u03b1-synuclein modulates target gene expression","volume":"533","author":"Soldner","year":"2016","journal-title":"Nature"},{"issue":"1","key":"10.1016\/j.gim.2025.101623_bib46","doi-asserted-by":"crossref","first-page":"6330","DOI":"10.1038\/s41467-022-33819-3","article-title":"Function of bidirectional sensitivity in the otolith organs established by transcription factor Emx2","volume":"13","author":"Ji","year":"2022","journal-title":"Nat Commun"}],"container-title":["Genetics in Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1098360025002709?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1098360025002709?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T23:25:01Z","timestamp":1786663501000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1098360025002709"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":46,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["S1098360025002709"],"URL":"https:\/\/doi.org\/10.1016\/j.gim.2025.101623","relation":{},"ISSN":["1098-3600"],"issn-type":[{"value":"1098-3600","type":"print"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"De novo rare EMX2 variants lead to idiopathic hypogonadotropic hypogonadism","name":"articletitle","label":"Article Title"},{"value":"Genetics in Medicine","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.gim.2025.101623","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of American College of Medical Genetics and Genomics.","name":"copyright","label":"Copyright"}],"article-number":"101623"}}