| Literature DB >> 32279304 |
Virginie Carmignac1,2, Sophie Nambot1,3,4,5, Daphné Lehalle3, Patrick Callier1,4,5, Stephanie Moortgat6, Valérie Benoit6, Jamal Ghoumid7,8, Bruno Delobel9, Thomas Smol8,10, Caroline Thuillier10, Cécile Zordan11, Sophie Naudion11, Thierry Bienvenu12,13,14, Renaud Touraine15, Francis Ramond15, Christiane Zweier16, André Reis16, Cornelia Kraus16, Mathilde Nizon17, Benjamin Cogné17, Alain Verloes18, Frédéric Tran Mau-Them1,4, Arthur Sorlin1,3,4, Thibaud Jouan1, Yannis Duffourd1,5, Emilie Tisserant1,5, Christophe Philippe1,4, Antonio Vitobello1,4, Julien Thevenon1,3,5, Laurence Faivre1,3,4,5, Christel Thauvin-Robinet1,4,5,19.
Abstract
X-linked intellectual disability (XLID) is a genetically heterogeneous condition involving more than 100 genes. To date, 35 pathogenic variants have been reported in the lysine specific demethylase 5C (KDM5C) gene. KDM5C variants are one of the major causes of moderate to severe XLID. Affected males present with short stature, distinctive facial features, behavioral disorders, epilepsy, and spasticity. For most of these variants, related female carriers have been reported, but phenotypic descriptions were poor. Here, we present clinical and molecular features of 19 females carrying 10 novel heterozygous variants affecting KDM5C function, including five probands with de novo variants. Four heterozygous females were asymptomatic. All affected individuals presented with learning disabilities or ID (mostly moderate), and four also had a language impairment mainly affecting expression. Behavioral disturbances were frequent, and endocrine disorders were more frequent in females. In conclusion, our findings provide evidence of the role of KDM5C in ID in females highlighting the increasing implication of XLID genes in females, even in sporadic affected individuals. Disease expression of XLID in females should be taken into consideration for genetic counseling.Entities:
Keywords: zzm321990KDM5C; X-linked intellectual disability; data-sharing; exome; females
Mesh:
Substances:
Year: 2020 PMID: 32279304 DOI: 10.1111/cge.13755
Source DB: PubMed Journal: Clin Genet ISSN: 0009-9163 Impact factor: 4.438