Search results for "medical genetics"

showing 10 items of 48 documents

9q33.3q34.11 microdeletion: new contiguous gene syndrome encompassing STXBP1, LMX1B and ENG genes assessed using reverse phenotyping

2016

International audience; The increasing use of array-CGH in malformation syndromes with intellectual disability could lead to the description of new contiguous gene syndrome by the analysis of the gene content of the microdeletion and reverse phenotyping. Thanks to a national and international call for collaboration by Achropuce and Decipher, we recruited four patients carrying de novo overlapping deletions of chromosome 9q33.3q34.11, including the STXBP1, the LMX1B and the ENG genes. We restrained the selection to these three genes because the effects of their haploinsufficency are well described in the literature and easily recognizable clinically. All deletions were detected by array-CGH …

0301 basic medicineMale[ SDV.MHEP.PED ] Life Sciences [q-bio]/Human health and pathology/PediatricsHaploinsufficiencycerebral hypomyelinationwest-syndromeBioinformaticsCraniofacial Abnormalities0302 clinical medicineIntellectual disabilitySTXBP1ChildGenetics (clinical)Nail patella syndromeGeneticsEndoglinSyndrome3. Good healthdevelopmental delayPhenotypeintellectual disabilityMedical geneticsFemaleChromosome DeletionHaploinsufficiencyChromosomes Human Pair 9medicine.medical_specialtyAdolescentLIM-Homeodomain ProteinsBiologyContiguous gene syndromeArticle03 medical and health sciencesMunc18 ProteinsGenetic linkageGeneticsmedicineHumansde-novo mutations[SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/PediatricsdiseaseEpilepsyinfantile epileptic encephalopathyassociationdeletionsmedicine.diseaseHuman genetics030104 developmental biologynail-patella syndrome030217 neurology & neurosurgeryTranscription Factors
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One NF1 Mutation may Conceal Another

2019

Neurofibromatosis type 1 (NF1) is an autosomal dominant disease with complete penetrance but high variable expressivity. NF1 is caused by loss-of-function mutations in the NF1 gene, a negative regulator of the RAS-MAPK pathway. The NF1 gene has one of the highest mutation rates in human disorders, which may explain the outbreak of independent de novo variants in the same family. Here, we report the co-occurrence of pathogenic variants in the NF1 and SPRED1 genes in six families with NF1 and Legius syndrome, using next-generation sequencing. In five of these families, we observed the co-occurrence of two independent NF1 variants. All NF1 variants were classified as pathogenic, according to t…

0301 basic medicineMutation ratemedicine.medical_specialtySPRED1congenital hereditary and neonatal diseases and abnormalities<i>SPRED1</i>lcsh:QH426-470[SDV]Life Sciences [q-bio]030105 genetics & heredityBiologyneurofibromatosis type 103 medical and health sciencesGeneticsmedicineNeurofibromatosisneoplasmsGenetics (clinical)Legius syndromeGeneticsMolecular pathologyAutosomal dominant traitmedicine.diseasePenetrance<i>NF1</i>eye diseases3. Good healthnervous system diseases[SDV] Life Sciences [q-bio]Legius syndromelcsh:Genetics030104 developmental biologyNF1Medical geneticsSPRED1 Genede novo variantGenes
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Improving the Management of Patients with Hearing Loss by the Implementation of an NGS Panel in Clinical Practice

2020

A cohort of 128 patients from 118 families diagnosed with non-syndromic or syndromic hearing loss (HL) underwent an exhaustive clinical evaluation. Molecular analysis was performed using targeted next-generation sequencing (NGS) with a custom panel that included 59 genes associated with non-syndromic HL or syndromic HL. Variants were prioritized according to the minimum allele frequency and classified according to the American College of Medical Genetics and Genomics guidelines. Variant(s) responsible for the disease were detected in a 40% of families including autosomal recessive (AR), autosomal dominant (AD) and X-linked patterns of inheritance. We identified pathogenic or likely pathogen…

0301 basic medicineOncologyAdultMalemedicine.medical_specialtyAdolescentlcsh:QH426-470Hearing lossHearing Loss Sensorineuralclinical evaluationPopulationGenomicsDiseaseDeafnessArticle03 medical and health sciences0302 clinical medicineInternal medicinemedicineHumansgeneticsmolecular analysiseducationChildAllele frequencyGenetics (clinical)hearing losseducation.field_of_studybusiness.industryInfant NewbornHigh-Throughput Nucleotide SequencingInfantMiddle Agedmedicine.diseaselcsh:Genetics030104 developmental biologyChild PreschoolCohortMedical geneticsSensorineural hearing lossFemalenext-generation sequencingmedicine.symptombusiness030217 neurology & neurosurgeryGenes
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Does breast carcinoma belong to the Lynch syndrome tumor spectrum? : Somatic mutational profiles vs. ovarian and colorectal carcinomas

2020

// Noora K. Porkka 1 , Alisa Olkinuora 1 , Teijo Kuopio 2 , 3 , Maarit Ahtiainen 4 , Samuli Eldfors 5 , Henrikki Almusa 5 , Jukka-Pekka Mecklin 6 , 7 , 8 and Paivi Peltomaki 1 1 Department of Medical and Clinical Genetics, University of Helsinki, Helsinki, Finland 2 Department of Pathology, Jyvaskyla Central Hospital, Jyvaskyla, Finland 3 Department of Biological and Environmental Science, University of Jyvaskyla, Jyvaskyla, Finland 4 Department of Education and Research, Jyvaskyla Central Hospital and University of Eastern Finland, Jyvaskyla, Finland 5 Institute for Molecular Medicine Finland, University of Helsinki, Helsinki, Finland 6 Faculty of Sport and Health Sciences, University of J…

0301 basic medicineOncologymedicine.medical_specialtyDNA mismatch repair3122 Cancersmedicine.disease_cause03 medical and health sciences0302 clinical medicineBreast cancerGermline mutationInternal medicinemedicinesomatic mutationbreast carcinomaLynchin oireyhtymäMSIperinnölliset tauditrintasyöpäbusiness.industryCancermedicine.diseaseLynch syndromedigestive system diseases3. Good health030104 developmental biologyLynch syndromeOncologysyöpägeenit030220 oncology & carcinogenesisMedical geneticsDNA mismatch repairsyöpätaudit3111 BiomedicinemutaatiotCarcinogenesisBreast carcinomabusinessResearch Paper
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WES/WGS Reporting of Mutations from Cardiovascular "Actionable" Genes in Clinical Practice: A Key Role for UMD Knowledgebases in the Era of Big Datab…

2016

International audience; High-throughput next-generation sequencing such as whole-exome and whole-genome sequencing are being rapidly integrated into clinical practice. The use of these techniques leads to the identification of secondary variants for which decisions about the reporting or not to the patient need to be made. The American College of Medical Genetics and Genomics recently published recommendations for the reporting of these variants in clinical practice for 56 "actionable" genes. Among these, seven are involved in Marfan Syndrome And Related Disorders (MSARD) resulting from mutations of the FBN1, TGFBR1 and 2, ACTA2, SMAD3, MYH11 and MYLK genes. Here, we show that mutations col…

0301 basic medicinemedicine.medical_specialtyKnowledge BasesGenomicsmarfan-syndrome[SDV.GEN.GH] Life Sciences [q-bio]/Genetics/Human genetics030105 genetics & heredityBiologycomputer.software_genreGenomeExAC03 medical and health sciencesAnnotationincidental findingsGeneticsmedicineHumanspathogenicityGenetic Predisposition to Diseasetgfbr2ExomegenomeESPGenetics (clinical)Exome sequencing[INFO.INFO-BI] Computer Science [cs]/Bioinformatics [q-bio.QM]variantsDatabasethoracic aortic-aneurysmsGenome HumanHigh-Throughput Nucleotide SequencingMYLKGenomicspredictionmutations3. Good healthMarfan syndrome030104 developmental biologydissection[SDV.GEN.GH]Life Sciences [q-bio]/Genetics/Human geneticsCardiovascular DiseasesMutationMedical geneticsIdentification (biology)LSDB[INFO.INFO-BI]Computer Science [cs]/Bioinformatics [q-bio.QM]computerexome
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Les modifications de pratique clinique liées à l’arrivée du séquençage haut débit dans le diagnostic génétique des maladies du développement

2018

Abstract Introduction The arrival of high-throughput sequencing (HTS) has led to a sweeping change in the diagnosis of developmental abnormalities (DA) with or without intellectual deficiency (ID). With the prospect of deploying these new technologies, two questions have been raised: the representations of HTS among geneticists and the costs incurred due to these analyses. Methods Geneticists attending a clinical genetics seminar were invited to complete a questionnaire. The statistical analysis was essentially descriptive and an analysis of costs was undertaken. Results Of those responding to the questionnaire, 48% had already prescribed exome analysis and 25% had already had the occasion …

0301 basic medicinemedicine.medical_specialtyMedical practice3. Good health03 medical and health sciences030104 developmental biologyFamily medicinePediatrics Perinatology and Child HealthmedicineCost analysisMedical geneticsGenomic medicineStatistical analysisPsychologyExomeArchives de Pédiatrie
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ISL1 is a major susceptibility gene for classic bladder exstrophy and a regulator of urinary tract development

2017

AbstractPreviously genome-wide association methods in patients with classic bladder exstrophy (CBE) found association with ISL1, a master control gene expressed in pericloacal mesenchyme. This study sought to further explore the genetics in a larger set of patients following-up on the most promising genomic regions previously reported. Genotypes of 12 markers obtained from 268 CBE patients of Australian, British, German Italian, Spanish and Swedish origin and 1,354 ethnically matched controls and from 92 CBE case-parent trios from North America were analysed. Only marker rs6874700 at the ISL1 locus showed association (p = 2.22 × 10−08). A meta-analysis of rs6874700 of our previous and prese…

0301 basic medicinemedicine.medical_specialtyPathologyMesenchymeUrinary systemOrganogenesisLIM-Homeodomain ProteinsLocus (genetics)030105 genetics & heredityBiologyPolymorphism Single Nucleotidebladder extrophyArticlePronephrosMesoderm03 medical and health sciencesMiceBEEC bladder extrophy urinary tract development ISL1GenotypemedicineAnimalsHumansProtein IsoformsGenetic Predisposition to DiseaseBEECUrinary TractGeneZebrafishGeneticsMultidisciplinaryBladder ExstrophyGene Expression Regulation DevelopmentalISL1medicine.diseaseEmbryo Mammalianurinary tract developmentBladder exstrophy030104 developmental biologymedicine.anatomical_structureReconstructive and regenerative medicine Radboud Institute for Molecular Life Sciences [Radboudumc 10]LarvaISL1Medical geneticsFemaleTranscription FactorsRare cancers Radboud Institute for Health Sciences [Radboudumc 9]Scientific Reports
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Genome-wide analysis reveals DNA methylation markers that vary with both age and obesity

2014

AbstractThe combination of the obesity epidemic and an aging population presents growing challenges for the healthcare system. Obesity and aging are major risk factors for a diverse number of diseases and it is of importance to understand their interaction and the underlying molecular mechanisms. Herein the authors examined the methylation levels of 27578 CpG sites in 46 samples from adult peripheral blood. The effect of obesity and aging was ascertained with general linear models. More than one hundred probes were correlated to aging, nine of which belonged to the KEGG group map04080. Additionally, 10 CpG sites had diverse methylation profiles in obese and lean individuals, one of which wa…

AdultGenetic Markersmedicine.medical_specialtyAgingAlpha-Ketoglutarate-Dependent Dioxygenase FTOGenome-wide association studyBiologyMicroarrayBioinformaticsEpigenesis GeneticGeneticsmedicineHumansEpigeneticsObesityKEGGTelomeraseAgedMedicinsk genetikGeneticsProteinsGeneral MedicineMethylationDNA MethylationMiddle Agedmedicine.diseaseObesityCpG siteDNA methylationMedical geneticsCpG IslandsFemaleEpigeneticsMedical GeneticsGenome-Wide Association Study
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Cardiomyopathy due to PRDM16 mutation: First description of a fetal presentation, with possible modifier genes

2020

PRDM16 (positive regulatory domain 16) is localized in the critical region for cardiomyopathy in patients with deletions of chromosome 1p36, as defined by Gajecka et al., American Journal of Medical Genetics, 2010, 152A, 3074-3083, and encodes a zinc finger transcription factor. We present the first fetal case of left ventricular non-compaction (LVNC) with a PRDM16 variant. The third-trimester obstetric ultrasound revealed a hydropic fetus with hydramnios and expanded hypokinetic heart. After termination of pregnancy, foetopathology showed a eutrophic fetus with isolated cardiomegaly. Endocardial fibroelastosis was associated with non-compaction of the myocardium of the left ventricle. Exom…

AdultHeart Defects CongenitalMalemedicine.medical_specialtyCardiomyopathyBiologyLabor PresentationGenetic HeterogeneityPregnancyExome SequencingGeneticsmedicineHumansMissense mutationGenetic Predisposition to DiseaseGenetics (clinical)Exome sequencingGeneticsFetusGenes ModifierGenetic heterogeneityInfant NewbornEndocardial fibroelastosisMiddle AgedFetal Presentationmedicine.diseasePedigreeDNA-Binding ProteinsMutationMedical geneticsFemaleCardiomyopathiesTranscription FactorsAmerican Journal of Medical Genetics Part C: Seminars in Medical Genetics
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Genetic and environmental contributions to the inverse association between specific autistic traits and experience seeking in adults

2015

Autistic traits are characterized by social and communication problems, restricted, repetitive and stereotyped patterns of behavior, interests and activities. The relation between autistic traits and personality characteristics is largely unknown. This study focused on the relation between five specific autistic traits measured with the abridged version of the Autism Spectrum Quotient ("social problems," "preference for routine," "attentional switching difficulties," "imagination impairments," "fascination for numbers and patterns") and Experience Seeking (ES) in a general population sample of adults, and subsequently investigated the genetic and environmental etiology between these traits.…

AdultMaleAutism-spectrum quotientmedicine.medical_specialtyInverse Associationmedia_common.quotation_subjectTwinsEnvironmentDevelopmental psychology03 medical and health sciencesCellular and Molecular NeuroscienceRisk-Taking0302 clinical medicinemedicineHumansSensation seekingPersonalityAttention0501 psychology and cognitive sciencesAutistic DisorderAssociation (psychology)Genetics (clinical)Netherlandsmedia_commonGeneticsSiblings05 social sciencesmedicine.diseasePreferenceSubstance abusePsychiatry and Mental healthAutistic traitsEtiologyMedical geneticsAutismFemalePsychology030217 neurology & neurosurgeryPersonality050104 developmental & child psychologyAmerican Journal of Medical Genetics Part B: Neuropsychiatric Genetics
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