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showing 10 items of 10255 documents

Genetic epidemiology of autosomal recessive hypercholesterolemia in Sicily: Identification by next-generation sequencing of a new kindred.

2017

Background Autosomal recessive hypercholesterolemia (ARH) is a rare inherited lipid disorder. In Sardinia, differently from other world regions, the mutated allele frequency is high. It is caused by mutations in the low-density lipoprotein receptor adaptor protein 1 gene. Fourteen different mutations have been reported so far; in Sardinia, 2 alleles (ARH1 and ARH2) explain most of the cases. Four ARH patients, all carriers of the ARH1 mutation, have been identified in mainland Italy and 2 in Sicily. Objective The objectives of the study were to improve the molecular diagnosis of familial hypercholesterolemia (FH) and to estimate the frequency of the ARH1 allele in 2 free-living Sicilian pop…

0301 basic medicineMaleSettore MED/09 - Medicina InternaEndocrinology Diabetes and MetabolismFamilial hypercholesterolemia030204 cardiovascular system & hematology0302 clinical medicineChildN-Glycosyl HydrolasesSicilyGeneticsAged 80 and overeducation.field_of_studyNutrition and DieteticsAllele frequencyHomozygoteHigh-Throughput Nucleotide SequencingAutosomal recessive hypercholesterolemiaMiddle AgedAutosomal Recessive HypercholesterolemiaSettore MED/26 - NeurologiaFemaleCardiology and Cardiovascular MedicineAdultAdolescentGenotypePopulationHypercholesterolemiaBiologyDNA sequencing03 medical and health sciencesYoung AdultARH1Internal MedicinemedicineHumansAlleleeducationGenotypingAllele frequencyAllelesAdaptor Proteins Signal TransducingAgedHeterozygous carrierSequence Analysis DNAmedicine.diseaseNGS-based gene panel030104 developmental biologyGenetic epidemiologyReceptors LDLJournal of clinical lipidology
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PDXK mutations cause polyneuropathy responsive to pyridoxal 5′‐phosphate supplementation

2019

OBJECTIVE: To identify disease-causing variants in autosomal recessive axonal polyneuropathy with optic atrophy and provide targeted replacement therapy. METHODS: We performed genome-wide sequencing, homozygosity mapping, and segregation analysis for novel disease-causing gene discovery. We used circular dichroism to show secondary structure changes and isothermal titration calorimetry to investigate the impact of variants on adenosine triphosphate (ATP) binding. Pathogenicity was further supported by enzymatic assays and mass spectroscopy on recombinant protein, patient-derived fibroblasts, plasma, and erythrocytes. Response to supplementation was measured with clinical validated rating sc…

0301 basic medicineMale[SDV.NEU.NB]Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC]/NeurobiologyLOCAL TRANSLATIONMedizinmedicine.disease_causeDISEASEchemistry.chemical_compound0302 clinical medicinepolineuropathyCinètica enzimàticaGene Regulatory NetworksPyridoxal phosphateChildPyridoxal KinaseAdenosine triphosphate (ATP)Research ArticlesAged 80 and overMutationGene Regulatory NetworkPLASMAAutosomal recessive axonal polyneuropathyDisease gene identificationPyridoxal kinase3. Good healthSettore MED/26 - NEUROLOGIANeuropaties perifèriquesTreatment OutcomePolyneuropathieNeurologyChild PreschoolPyridoxal PhosphateRELIABILITYVitamin B ComplexFemaleLife Sciences & BiomedicinePolyneuropathyHumanResearch ArticleAdultAdolescentPDXKClinical NeurologyCHARCOT-MARIE-TOOTHCHARCOT-MARIE-TOOTH CMT NEUROPATHY SCORE LOCAL TRANSLATION DISEASE RELIABILITY; MECHANISMS DISCOVERY FRAMEWORK KINASE PLASMAMECHANISMS03 medical and health sciencesPolyneuropathiesAtrophy[SDV.BBM.GTP]Life Sciences [q-bio]/Biochemistry Molecular Biology/Genomics [q-bio.GN]KINASEmedicineHumansCMT NEUROPATHY SCOREPDXK mutationsPyridoxalDietary SupplementAgedPeripheral neuropathiesScience & Technology[SCCO.NEUR]Cognitive science/NeuroscienceEnzyme kineticsNeurosciencesFRAMEWORKmedicine.diseaseMolecular biology030104 developmental biologychemistryDISCOVERYDietary SupplementsMutationNeurosciences & NeurologyNeurology (clinical)Adenosine triphosphate030217 neurology & neurosurgeryAnnals of Neurology
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Retrospective Analysis of Checkpoint Inhibitor Therapy-Associated Cases of Bullous Pemphigoid From Six German Dermatology Centers

2021

Immune-related adverse events (irAEs) are a class-effect of checkpoint inhibitors (CIs). The development of a Bullous pemphigoid (BP)-like blistering disease, driven by autoantibodies against the hemidesmosomal protein BP180, is a potentially serious irAE whose incidence seems to be increasing. We therefore set out to characterize the clinical and (immuno)histopathological features and treatment responses of cases of BP which developed during or after CI therapy collated in six German tertiary referral centers between 2014 and 2018. We identified twelve cases of BP which emerged during and/or after CI therapy. The time interval between the initiation of CI therapy and the diagnosis of BP wa…

0301 basic medicineMalelcsh:Immunologic diseases. Allergymedicine.medical_specialtyPD-1 - PD-L1 axisautoantibodiesImmune checkpoint inhibitorsImmunologypemphigoid diseaseIpilimumabPembrolizumabDermatology030207 dermatology & venereal diseases03 medical and health sciences0302 clinical medicineAntineoplastic Agents ImmunologicalAdrenal Cortex HormonesInternal medicineGermanyNeoplasmsPemphigoid BullousmedicineHumansImmunology and AllergyipilimumabAdverse effectImmune Checkpoint InhibitorsAgedRetrospective StudiesOriginal ResearchAged 80 and overnivolumabbusiness.industryIncidence (epidemiology)autoimmunityAutoantibodyMiddle Agedmedicine.disease030104 developmental biologyFemaleBullous pemphigoidpembrolizumabNivolumabbusinesslcsh:RC581-607checkpoint inhibitorsmedicine.drugFrontiers in Immunology
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Angiotensin II type II receptors and colonic dysmotility in 2,4-dinitrofluorobenzenesulfonic acid-induced colitis in rats

2016

Background: Angiotensin II (Ang II), the main peptide of the renin-angiotensin system (RAS), has been suggested to be involved in inflammatory bowel diseases. Since RAS has emerged as gut motility regulator, and dysmotility is associated with intestinal inflammation, our objective was to investigate in rat 2,4-dinitrobenzenesulfonic acid (DNBS)-induced colitis the functionality of RAS and its contribution to colonic motor alterations. Methods: The effects of Ang II on the longitudinal colonic muscular contractility of control and DNBS-treated rats were characterized in vitro. Transcripts encoding for Ang II receptors were investigated by RT-PCR. Key Results: Inflamed preparations showed a l…

0301 basic medicineMalemedicine.medical_specialtyAngiotensin receptormedicine.drug_classColonPhysiologyInflammationAT2 receptorReceptor Angiotensin Type 2Bowel inflammationEndocrine and Autonomic SystemContractilityRenin-Angiotensin System03 medical and health sciences0302 clinical medicineInternal medicinemedicineAnimalsRats WistarReceptorAngiotensin II receptor type 1Endocrine and Autonomic SystemsChemistryAT1 receptorAngiotensin IIMuscle contractilityGastroenterologyMuscle SmoothNitric oxideReceptor antagonistColitisAngiotensin II030104 developmental biologyEndocrinologyLosartancardiovascular system030211 gastroenterology & hepatologyDinitrofluorobenzenemedicine.symptomGastrointestinal Motilityhormones hormone substitutes and hormone antagonistsmedicine.drugMuscle Contraction
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Opposite effects of dopamine on the mechanical activity of circular and longitudinal muscle of human colon.

2020

Background Because dopamine (DA) has gained increasing evidence as modulator of gut motility, we aimed to characterize dopaminergic response in human colon, evaluating function and distribution of dopamine receptors in circular vs longitudinal muscle strips. Methods Mechanical responses to DA and dopaminergic agonists on slow phasic contractions and on basal tone were examined in vitro as changes in isometric tension. RT-PCR was used to reveal the distribution of dopaminergic receptors. Key results In spontaneous active circular muscle, DA induced an increase in the amplitude of slow phasic contractions and of the basal tone, via activation of D1-like receptors. DA contractile responses wer…

0301 basic medicineMalemedicine.medical_specialtyContraction (grammar)intestinal motilityPhysiologyColonDopamineIsometric exerciseSettore BIO/09 - FisiologiaReceptors Dopamine03 medical and health sciences0302 clinical medicineDopamineInternal medicinemedicineHumansReceptordopaminergic receptors human colonAgedAged 80 and overbiologyPhospholipase CEndocrine and Autonomic SystemsChemistryDopaminergicGastroenterologyMuscle SmoothMiddle AgedNitric oxide synthase030104 developmental biologyEndocrinologyDopamine receptorDopamine Agonistsbiology.proteinFemalecircular and longitudinal muscle030217 neurology & neurosurgerymedicine.drugMuscle ContractionNeurogastroenterology and motility : the official journal of the European Gastrointestinal Motility SocietyREFERENCES
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Amino Acid Polymorphisms in Hla Class II Differentiate Between Thyroid and Polyglandular Autoimmunity.

2019

Abstract Context The structure of the human leucocyte antigen (HLA) peptide-binding clefts strongly contributes to monoglandular and polyglandular autoimmunity (AP). Objective To investigate the impact of amino acid polymorphisms on the peptide-binding interactions within HLA class II and its association with AP. Design Immunogenetic study. Setting Tertiary referral center for autoimmune endocrine diseases. Subjects 587 subjects with AP, autoimmune thyroid disease (AITD), type 1 diabetes (T1D), and healthy unrelated controls were typed for HLA class II. Methods Amino acids within the peptide binding cleft that are encoded by HLA class II exon 2 were listed for all codon positions in all sub…

0301 basic medicineMalemedicine.medical_specialtyEndocrinology Diabetes and MetabolismClinical Biochemistry030209 endocrinology & metabolismPeptide bindingImmunogeneticsHuman leukocyte antigenBiologymedicine.disease_causeBiochemistryAutoimmunityDiagnosis Differential03 medical and health sciencesExon0302 clinical medicineEndocrinologyInternal medicinemedicineHumansGenetic Predisposition to DiseaseAlleleAmino AcidsPolyendocrinopathies AutoimmunePolymorphism GeneticBiochemistry (medical)ThyroidHistocompatibility Antigens Class IIThyroiditis AutoimmuneAutoimmune polyendocrinopathyPrognosis030104 developmental biologyEndocrinologymedicine.anatomical_structureDiabetes Mellitus Type 1Case-Control StudiesFemaleBiomarkersFollow-Up StudiesThe Journal of clinical endocrinology and metabolism
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Performance evaluation of a newly developed molecular assay for the accurate diagnosis of gastroenteritis associated with norovirus of genogroup II

2018

The performance of a newly proposed fully automated cassette-based sample-to-results solution for norovirus (NoV) detection, InGenius Norovirus ELITe MGB®, was evaluated. A total of 120 selected archival stool samples from children hospitalized for acute gastroenteritis were used to compare the results to a reference real-time RT-PCR. The InGenius NoV assay showed optimal diagnostic accuracy (sensitivity, 100%; specificity, 95.7%) and was able to correctly detect the entire wide panel of epidemiologically relevant genotypes tested. These preliminary results suggest that the InGenius NoV assay can be recommended as a valuable method for accurate diagnosis of NoV GII infection in epidemic and…

0301 basic medicineMalemedicine.medical_specialtySettore MED/07 - Microbiologia E Microbiologia ClinicaAdolescentGenotype030106 microbiologyDiagnostic accuracyBiologymedicine.disease_causeReal-Time Polymerase Chain ReactionSensitivity and SpecificityFeces03 medical and health sciencesMedical microbiologyVirologyGenotypemedicineHumansChildCaliciviridae InfectionsNoroviruCaliciviridae InfectionGastroenteritiReverse Transcriptase Polymerase Chain ReactionNorovirusInfantGeneral MedicineAcute gastroenteritisbacterial infections and mycosesVirologyGastroenteritis030104 developmental biologyCaliciviridae InfectionsFully automatedChild PreschoolNorovirusFeceFemaleHuman
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Renal tubular epithelial cell-derived BAFF expression mediates kidney damage and correlates with activity of proliferative lupus nephritis in mouse a…

2017

B-cell activating factor of the tumour necrosis factor family (BAFF) is a cytokine, mainly produced by hematopoietic cells (e.g. monocytes/macrophages, dendritic cells), indispensable for B-cell maturation. The BLISS studies have demonstrated that blocking BAFF by the human monoclonal antibody belimumab is a valuable therapeutic approach in patients with clinically and serologically active systemic lupus erythematosus (SLE). However, the defined sources of BAFF, which contributes to SLE, are still unclear. Recent findings show that BAFF expression is not restricted to myeloid cells. Since lupus nephritis is the main cause of morbidity and mortality for SLE patients, the aim of this study wa…

0301 basic medicineMalemedicine.medical_treatmentLupus nephritisAntibodies Monoclonal HumanizedKidneySeverity of Illness IndexPathogenesis03 medical and health sciencesMice0302 clinical medicinestomatognathic systemRheumatologyimmune system diseasesB-Cell Activating FactormedicineAnimalsHumansLupus Erythematosus Systemicskin and connective tissue diseasesB-cell activating factorAutocrine signallingRetrospective StudiesB-Lymphocytesbusiness.industryTumor Necrosis Factor-alphaEpithelial Cellsmedicine.diseaseBelimumabLupus Nephritisstomatognathic diseasesHaematopoiesis030104 developmental biologyCytokineReceptors Granulocyte-Macrophage Colony-Stimulating FactorImmunologyCytokinesTumor necrosis factor alphaFemaleKidney DiseasesbusinessImmunosuppressive Agents030215 immunologymedicine.drugLupus
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A Dirichlet Autoregressive Model for the Analysis of Microbiota Time-Series Data

2021

Growing interest in understanding microbiota dynamics has motivated the development of different strategies to model microbiota time series data. However, all of them must tackle the fact that the available data are high-dimensional, posing strong statistical and computational challenges. In order to address this challenge, we propose a Dirichlet autoregressive model with time-varying parameters, which can be directly adapted to explain the effect of groups of taxa, thus reducing the number of parameters estimated by maximum likelihood. A strategy has been implemented which speeds up this estimation. The usefulness of the proposed model is illustrated by application to a case study.

0301 basic medicineMathematical optimizationMultidisciplinaryArticle SubjectGeneral Computer ScienceComputer scienceMaximum likelihoodQA75.5-76.9501 natural sciencesDirichlet distribution010104 statistics & probability03 medical and health sciencessymbols.namesake030104 developmental biologyAutoregressive modelElectronic computers. Computer sciencesymbols0101 mathematicsTime seriesComplexity
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Extracellular Hsp70 Enhances Mesoangioblast Migration via an Autocrine Signaling Pathway

2017

Mouse mesoangioblasts are vessel-associated progenitor stem cells endowed with the ability of multipotent mesoderm differentiation. Therefore, they represent a promising tool in the regeneration of injured tissues. Several studies have demonstrated that homing of mesoangioblasts into blood and injured tissues are mainly controlled by cytokines/chemokines and other inflammatory factors. However, little is known about the molecular mechanisms regulating their ability to traverse the extracellular matrix (ECM). Here, we demonstrate that membrane vesicles released by mesoangioblasts contain Hsp70, and that the released Hsp70 is able to interact by an autocrine mechanism with Toll-like receptor …

0301 basic medicineMesoangioblastPhysiologyChemistryClinical BiochemistryCell migrationCell BiologyCell biologyExtracellular matrix03 medical and health sciences030104 developmental biologyExtracellularStem cellSignal transductionAutocrine signallingPI3K/AKT/mTOR pathwayJournal of Cellular Physiology
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