Search results for "Agenesis of the corpus callosum"

showing 8 items of 8 documents

Heterozygous Variants in KDM4B Lead to Global Developmental Delay and Neuroanatomical Defects

2020

International audience; KDM4B is a lysine-specific demethylase with a preferential activity on H3K9 tri/di-methylation (H3K9me3/2)-modified histones. H3K9 tri/di-demethylation is an important epigenetic mechanism responsible for silencing of gene expression in animal development and cancer. However, the role of KDM4B on human development is still poorly characterized. Through international data sharing, we gathered a cohort of nine individuals with mono-allelic de novo or inherited variants in KDM4B. All individuals presented with dysmorphic features and global developmental delay (GDD) with language and motor skills most affected. Three individuals had a history of seizures, and four had a…

0301 basic medicineMaleJumonji Domain-Containing Histone Demethylases[SDV]Life Sciences [q-bio]Developmental DisabilitiesCorpus callosumHippocampusEpigenesis GeneticHistonesMice0302 clinical medicineNeurodevelopmental disorderPolymicrogyriaGlobal developmental delayAgenesis of the corpus callosumGenetics (clinical)BrainMagnetic Resonance Imaging[SDV] Life Sciences [q-bio]intellectual disabilityBrain sizeFemaledysmorphic hippocampiSignal TransductionHeterozygoteheterozygous variantglobal developmental delayBiologyNervous System MalformationsMethylation03 medical and health sciencesSeizuresReportKDM4BGeneticsmedicineAnimalsHumansneurodevelopmental disorder.Dentate gyrusGenetic VariationJMJD2Bmedicine.diseaseneurodevelopmental disorder030104 developmental biologyagenesis of the corpus callosumNeuroscienceProtein Processing Post-Translational030217 neurology & neurosurgeryVentriculomegalyAmerican journal of human genetics
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Both rare and de novo copy number variants are prevalent in agenesis of the corpus callosum but not in cerebellar hypoplasia or polymicrogyria.

2013

Agenesis of the corpus callosum (ACC), cerebellar hypoplasia (CBLH), and polymicrogyria (PMG) are severe congenital brain malformations with largely undiscovered causes. We conducted a large-scale chromosomal copy number variation (CNV) discovery effort in 255 ACC, 220 CBLH, and 147 PMG patients, and 2,349 controls. Compared to controls, significantly more ACC, but unexpectedly not CBLH or PMG patients, had rare genic CNVs over one megabase (p = 1.48×10−3; odds ratio [OR] = 3.19; 95% confidence interval [CI] = 1.89–5.39). Rare genic CNVs were those that impacted at least one gene in less than 1% of the combined population of patients and controls. Compared to controls, significantly more AC…

AdultMaleCancer ResearchMicrocephalycongenital hereditary and neonatal diseases and abnormalitiesAdolescentDNA Copy Number Variationslcsh:QH426-470Developmental DisabilitiesPopulationGenome-wide association studyBiologyNervous System MalformationsCorpus callosumPolymorphism Single Nucleotide03 medical and health sciences0302 clinical medicineCerebellummental disordersGeneticsPolymicrogyriamedicineHumansCopy-number variationChildAgenesis of the corpus callosumeducationMolecular BiologyGenetics (clinical)Ecology Evolution Behavior and SystematicsExome sequencing030304 developmental biologyGenetics0303 health scienceseducation.field_of_studyGenome HumanInfant NewbornInfantMiddle Agedmedicine.disease3. Good healthMalformations of Cortical Developmentlcsh:GeneticsChild PreschoolFemaleAgenesis of Corpus Callosum030217 neurology & neurosurgeryResearch ArticleGenome-Wide Association StudyPLoS Genetics
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A novel L1CAM mutation in a fetus detected by prenatal diagnosis

2010

X-linked hydrocephalus is due to mutations in the L1 neuronal cell adhesion molecule (L1CAM) gene. L1 protein plays a key role in neurite outgrowth, axonal guidance, and pathfinding during the development of the nervous system. We report on a familial case diagnosed by prenatal ultrasonographic examination, with cerebellar hypoplasia, agenesis of the corpus callosum, and the bilateral overlapping of the second and third fingers of the hand. Sequencing of the L1CAM gene showed a novel missense mutation in exon 14: transition of a guanine to cytosine at position 1777 (c.1777G > C), which led to an amino acid change of alanine to proline at position 593 (Ala593Pro) in the sixth immunoglobulin …

Adultmedicine.medical_specialtyPathologyL1Neural Cell Adhesion Molecule L1Prenatal diagnosismedicine.disease_causeL1CAM L1-desease prenatal diagnosis hydrocephalus HSAS CRASH syndromeExonSettore MED/38 - Pediatria Generale E SpecialisticaPregnancyPrenatal DiagnosisInternal medicinemedicineHumansPoint MutationMissense mutationAgenesis of the corpus callosumUltrasonographyMutationbusiness.industrymedicine.diseasePedigreeFetal DiseasesEndocrinologyKaryotypingPediatrics Perinatology and Child HealthFemaleNeural cell adhesion moleculeCerebellar hypoplasia (non-human)businessHydrocephalus
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CNS involvement in OFD1 syndrome: a clinical, molecular, and neuroimaging study

2014

Background Oral-facial-digital type 1 syndrome (OFD1; OMIM 311200) belongs to the expanding group of disorders ascribed to ciliary dysfunction. With the aim of contributing to the understanding of the role of primary cilia in the central nervous system (CNS), we performed a thorough characterization of CNS involvement observed in this disorder. Methods A cohort of 117 molecularly diagnosed OFD type I patients was screened for the presence of neurological symptoms and/or cognitive/behavioral abnormalities on the basis of the available information supplied by the collaborating clinicians. Seventy-one cases showing CNS involvement were further investigated through neuroimaging studies and neur…

Central nervous systemNeuroimagingNeuropsychological TestsPharmacologyBioinformaticsSettore MED/03 - GENETICA MEDICACiliopathiesCohort Studies03 medical and health sciences0302 clinical medicineNeuroimagingCentral Nervous System DiseasesmedicineHumansGenetics(clinical)Pharmacology (medical)Orofaciodigital type 1Ciliopathies; Neurodevelopmental phenotype; Neuroimaging; OFD1; Central Nervous System Diseases; Cohort Studies; Female; Humans; Magnetic Resonance Imaging; Mutation; Neuropsychological Tests; Orofaciodigital Syndromes; Medicine (all); Genetics (clinical); Pharmacology (medical)Agenesis of the corpus callosumGenetics (clinical)030304 developmental biologyMedicine(all)0303 health sciencesbusiness.industryMedicine (all)ResearchCiliumNeuropsychologyCognitionGeneral MedicineOrofaciodigital Syndromesmedicine.diseasecentral nervous systemMagnetic Resonance ImagingPorencephalyCiliopathies3. Good healthmedicine.anatomical_structureMutationFemaleNeurodevelopmental phenotypeOFD1business030217 neurology & neurosurgeryOrphanet Journal of Rare Diseases
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Osseous oral hyaline ring granuloma mimicking a mandible tumor in a child with congenital agenesis of the corpus callosum

2017

Background Hyaline ring granuloma (HRG) of the oral cavity is an uncommon disorder considered to be a foreign-body reaction resulting from implantation of food vegetable particles. Microscopically, it is characterized by the presence of structures of hyaline rings in an inflamed fibrous tissue background, which contains multinucleated giant cells. Material and methods We present the case of a 4-year-old boy diagnosed with a mandible osseous HRG, which showed clinical and tomographic aspects suggestive of an aggressive bone tumor. Results The patient underwent surgical exploration and histopathologic analysis showed fragments composed predominantly of widespread dense connective tissue with …

Dense connective tissuePathologymedicine.medical_specialtyOdontologíaCase Report03 medical and health sciences0302 clinical medicineEosinophilicMedicineAgenesis of the corpus callosumGeneral DentistryHyalineOral Medicine and Pathologybusiness.industryMandible030206 dentistryAnatomy:CIENCIAS MÉDICAS [UNESCO]medicine.diseaseCiencias de la saludGiant cell030220 oncology & carcinogenesisGranulomaUNESCO::CIENCIAS MÉDICASChronic Inflammatory InfiltratebusinessJournal of Clinical and Experimental Dentistry
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Novel mutations including deletions of the entire OFD1 gene in 30 families with type 1 orofaciodigital syndrome : a study of the extensive clinical v…

2012

OFD1, now recognized as a ciliopathy, is characterized by malformations of the face, oral cavity and digits, and is transmitted as an X-linked condition with lethality in males. Mutations in OFD1 also cause X-linked Joubert syndrome (JBTS10) and Simpson-Golabi-Behmel syndrome type 2 (SGBS2). We have studied 55 sporadic and six familial cases of suspected OFD1. Comprehensive mutation analysis in OFD1 revealed mutations in 37 female patients from 30 families; 22 mutations have not been previously described including two heterozygous deletions spanning OFD1 and neighbouring genes. Analysis of clinical findings in patients with mutations revealed that oral features are the most reliable diagnos…

GeneticsMutationMedizinBiologymedicine.diseasemedicine.disease_causePhenotypeX-inactivationJoubert syndromeCiliopathyGeneticsmedicineMutation testingAgenesis of the corpus callosumGenetics (clinical)Ventriculomegaly
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NFIB Haploinsufficiency Is Associated with Intellectual Disability and Macrocephaly

2018

The nuclear factor I (NFI) family of transcription factors play an important role in normal development of multiple organs. Three NFI family members are highly expressed in the brain, and deletions or sequence variants in two of these, NFIA and NFIX, have been associated with intellectual disability (ID) and brain malformations. NFIB, however, has not previously been implicated in human disease. Here, we present a cohort of 18 individuals with mild ID and behavioral issues who are haploinsufficient for NFIB. Ten individuals harbored overlapping microdeletions of the chromosomal 9p23-p22.2 region, ranging in size from 225 kb to 4.3 Mb. Five additional subjects had point sequence variations c…

Male0301 basic medicinechromosome 9p23Medical and Health SciencesCorpus CallosumCohort StudiesMice2.1 Biological and endogenous factorsMegalencephalyAetiologyChildAgenesis of the corpus callosumGenetics (clinical)PediatricGenetics & HeredityCerebral CortexMice KnockoutGeneticsSingle Nucleotidenuclear factor IBiological SciencesNFIBNFIXdevelopmental delayMental HealthNFIBCodon NonsenseNFIAintellectual disabilityChild Preschoolchromosome 9p22.3NeurologicalSpeech delayFemalemedicine.symptomHaploinsufficiencyAdultAdolescentKnockoutIntellectual and Developmental Disabilities (IDD)[SDV.GEN.GH] Life Sciences [q-bio]/Genetics/Human geneticsBiologymacrocephalyPolymorphism Single NucleotideArticleYoung Adult03 medical and health sciencesRare DiseasesBehavioral and Social ScienceGeneticsmedicinemegalencephalyAnimalsHumansPolymorphismCodonPreschoolNeurosciencesMacrocephalymedicine.diseaseBrain DisordershaploinsufficiencyNFI Transcription Factors030104 developmental biologyNonsense[SDV.GEN.GH]Life Sciences [q-bio]/Genetics/Human geneticsbiology.proteinagenesis of the corpus callosumAmerican journal of human genetics
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Oral findings in Midline Syndrome: a case report and literature review

2010

We describe a female patient with a midline syndrome. The patient presents agenesis of the corpus callosum, encephalocele, iris coloboma, hypertelorism, submucosal cleft palate and dental anomalies. Despite being very characteristic, her phenotypical traits do not coincide exactly with those reported to date in the literature. The karyotype and the molecular cytogenetic study do not show mutations. We identify the presence of dental anomalies in the mother and other family members, not being identified MSX1 and PAX9 mutations that could the related with their etiology. Despite the fact that dental agenesis has been related to a large number of other malformation syndromes and congenital con…

medicine.medical_specialtyIrisEncephalocele03 medical and health sciences0302 clinical medicinestomatognathic systemMidline syndromeDental abnormalitiesmedicineHumansAbnormalities MultipleHypertelorismChildAgenesis of the corpus callosumGeneral DentistryEncephalocele030304 developmental biology0303 health sciencesHypertelorismTooth Abnormalitiesbusiness.industryDental agenesisSyndrome:CIENCIAS MÉDICAS [UNESCO]medicine.diseaseDermatologyIris coloboma3. Good healthSurgeryCleft PalateColobomastomatognathic diseasesPhenotypeOtorhinolaryngologyUNESCO::CIENCIAS MÉDICASEtiologyFemaleSurgeryMalformacionsAgenesis of Corpus Callosummedicine.symptombusinessMalformacions dentalsHuman abnormalitiesPAX9030217 neurology & neurosurgery
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