
pmid: 29861108
pmc: PMC5992133
Next-generation sequencing is a powerful tool for the discovery of genes related to neurodevelopmental disorders (NDDs). Here, we report the identification of a distinct syndrome due to de novo or inherited heterozygous mutations in Tousled-like kinase 2 (TLK2) in 38 unrelated individuals and two affected mothers, using whole-exome and whole-genome sequencing technologies, matchmaker databases, and international collaborations. Affected individuals had a consistent phenotype, characterized by mild-borderline neurodevelopmental delay (86%), behavioral disorders (68%), severe gastro-intestinal problems (63%), and facial dysmorphism including blepharophimosis (82%), telecanthus (74%), prominent nasal bridge (68%), broad nasal tip (66%), thin vermilion of the upper lip (62%), and upslanting palpebral fissures (55%). Analysis of cell lines from three affected individuals showed that mutations act through a loss-of-function mechanism in at least two case subjects. Genotype-phenotype analysis and comparison of computationally modeled faces showed that phenotypes of these and other individuals with loss-of-function variants significantly overlapped with phenotypes of individuals with other variant types (missense and C-terminal truncating). This suggests that haploinsufficiency of TLK2 is the most likely underlying disease mechanism, leading to a consistent neurodevelopmental phenotype. This work illustrates the power of international data sharing, by the identification of 40 individuals from 26 different centers in 7 different countries, allowing the identification, clinical delineation, and genotype-phenotype evaluation of a distinct NDD caused by mutations in TLK2.
Tousled-like ; Facial Averaging ; Haploinsufficiency ; Intellectual Disability ; Kinase, Male, Kinase, Biomedical and clinical sciences, Messenger, Intellectual disability, Medizin, Inheritance Patterns, Haploinsufficiency, Protein Kinases/genetics, Neurodevelopmental Disorders/genetics, Medical and Health Sciences, Translocation, Genetic, Loss of Function Mutation/genetics, Loss of Function Mutation, 2.1 Biological and endogenous factors, Genetics(clinical), Facial averaging, Aetiology, Child, Genetics & Heredity, Tousled-like, Biological Sciences, Deciphering Developmental Disorders Study, Biological sciences, intellectual disability, Child, Preschool, Female, facial averaging, Biotechnology, EMC NIHES-01-50-01-A, Adult, GENES, Adolescent, kinase, Bioinformatics and Computational Biology, 610, Translocation, Cell Line, Young Adult, Genetic, Clinical Research, Report, Genetics, Humans, RNA, Messenger, Preschool, Genetic Association Studies, Biomedical and Clinical Sciences, Radboudumc 7: Neurodevelopmental disorders DCMN: Donders Center for Medical Neuroscience, Base Sequence, TOUSLED-LIKE KINASES, MUTATIONS, Human Genome, Neurosciences, Health sciences, Facies, Infant, Radboudumc 9: Rare cancers RIHS: Radboud Institute for Health Sciences, Brain Disorders, Inheritance Patterns/genetics, haploinsufficiency, Neurodevelopmental Disorders, RNA, Messenger/genetics, Human Genetics - Radboud University Medical Center, Protein Kinases, ddc: ddc:
Tousled-like ; Facial Averaging ; Haploinsufficiency ; Intellectual Disability ; Kinase, Male, Kinase, Biomedical and clinical sciences, Messenger, Intellectual disability, Medizin, Inheritance Patterns, Haploinsufficiency, Protein Kinases/genetics, Neurodevelopmental Disorders/genetics, Medical and Health Sciences, Translocation, Genetic, Loss of Function Mutation/genetics, Loss of Function Mutation, 2.1 Biological and endogenous factors, Genetics(clinical), Facial averaging, Aetiology, Child, Genetics & Heredity, Tousled-like, Biological Sciences, Deciphering Developmental Disorders Study, Biological sciences, intellectual disability, Child, Preschool, Female, facial averaging, Biotechnology, EMC NIHES-01-50-01-A, Adult, GENES, Adolescent, kinase, Bioinformatics and Computational Biology, 610, Translocation, Cell Line, Young Adult, Genetic, Clinical Research, Report, Genetics, Humans, RNA, Messenger, Preschool, Genetic Association Studies, Biomedical and Clinical Sciences, Radboudumc 7: Neurodevelopmental disorders DCMN: Donders Center for Medical Neuroscience, Base Sequence, TOUSLED-LIKE KINASES, MUTATIONS, Human Genome, Neurosciences, Health sciences, Facies, Infant, Radboudumc 9: Rare cancers RIHS: Radboud Institute for Health Sciences, Brain Disorders, Inheritance Patterns/genetics, haploinsufficiency, Neurodevelopmental Disorders, RNA, Messenger/genetics, Human Genetics - Radboud University Medical Center, Protein Kinases, ddc: ddc:
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