Search results for "ADE"

showing 10 items of 15269 documents

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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Seafood Consumption, Omega-3 Fatty Acids Intake, and Life-Time Prevalence of Depression in the PREDIMED-Plus Trial.

2018

Background: The aim of this analysis was to ascertain the type of relationship between fish and seafood consumption, omega-3 polyunsaturated fatty acids (&omega

0301 basic medicineMaleeicosapentaenoic acidmodelos logísticoshumanosdepresiónencuestas sobre dietas0302 clinical medicineClinical trialsSurveys and QuestionnairesOdds RatioPrevalence030212 general & internal medicineDepressió psíquicamediana edadDietoteràpia2. Zero hungerancianoNutrition and Dieteticsintegumentary systemdietaDepressionEuropean researchFatty AcidsprevalenciaFishesfood and beveragesMiddle Ageddocosahexaenoic acidcociente de probabilidades relativasMental depressiondepressionChristian ministryFemaleomega-3lcsh:Nutrition. Foods and food supplyEuropean Regional Development Fundlcsh:TX341-641Àcids grassos omega-3Diet SurveysArticle03 medical and health sciencesalimentos del marDepression (economics)pecestrastorno depresivoEnvironmental healthPolitical scienceFatty Acids Omega-3ingesta energéticaOmega-3 fatty acidsAnimalsHumansácidos grasosAgedConsumption (economics)fishconducta alimentariaDepressive Disorder030109 nutrition & dieteticsDiet therapyLife timeFeeding BehaviorPredimedDietCross-Sectional StudiesLogistic ModelsSeafoodanimalesEnergy IntakeFood Scienceestudios transversalesAssaigs clínicsNutrients
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A multicentre, phase IIa study of zolbetuximab as a single agent in patients with recurrent or refractory advanced adenocarcinoma of the stomach or l…

2019

Abstract Background Claudin 18.2 (CLDN18.2) is physiologically confined to gastric mucosa tight junctions; however, upon malignant transformation, perturbations in cell polarity lead to CLDN18.2 epitopes being exposed on the cancer cell surface. The first-in-class monoclonal antibody, zolbetuximab (formerly known as IMAB362), binds to CLDN18.2 and can induce immune-mediated lysis of CLDN18.2-positive cells. Patients and methods Patients with advanced gastric, gastro-oesophageal junction (GEJ) or oesophageal adenocarcinomas with moderate-to-strong CLDN18.2 expression in ≥50% of tumour cells received zolbetuximab intravenously every 2 weeks for five planned infusions. At least three patients …

0301 basic medicineMalemedicine.medical_specialtyCLDN18.2Drug-Related Side Effects and Adverse ReactionsEsophageal NeoplasmsNauseagastro-oesophageal junction adenocarcinomaMedizinAdenocarcinomaGastroenterology03 medical and health sciences0302 clinical medicineStomach NeoplasmsInternal medicineGastrointestinal TumorsmedicineHumansProgression-free survivalAgedbusiness.industryStomachgastric cancerCancerAntibodies MonoclonalHematologyOriginal ArticlesMiddle Agedmedicine.diseaseddc:IMAB362030104 developmental biologymedicine.anatomical_structureTreatment OutcomeOncologyTolerability030220 oncology & carcinogenesisCohortVomitingAdenocarcinomaFemaleEsophagogastric Junctionmedicine.symptomzolbetuximabNeoplasm Recurrence Localbusiness
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Unexpected subcellular distribution of a specific isoform of the Coxsackie and adenovirus receptor, CAR-SIV, in human pancreatic beta cells

2018

Aims/hypothesis: The Coxsackie and adenovirus receptor (CAR) is a transmembrane cell-adhesion protein that serves as an entry receptor for enteroviruses and may be essential for their ability to infect cells. Since enteroviral infection of beta cells has been implicated as a factor that could contribute to the development of type 1 diabetes, it is often assumed that CAR is displayed on the surface of human beta cells. However, CAR exists as multiple isoforms and it is not known whether all isoforms subserve similar physiological functions. In the present study, we have determined the profile of CAR isoforms present in human beta cells and monitored the subcellular localisation of the princi…

0301 basic medicineMaleviruksetEndocrinology Diabetes and MetabolismInsulin-Secreting CellsProtein IsoformsReceptorChildProinsulinEnterovirusMicroscopy ConfocalChemistryNuclear ProteinsImmunogold labellingMiddle AgedFlow CytometryImmunohistochemistryTransmembrane protein3. Good healthCell biologyEndocrinologieenteroviruksetMédecine interneProtein interacting with C-kinase 1 (PICK1)medicine.anatomical_structureChild PreschoolCoxsackievirus BFemalePancreasPICK1Gene isoformBeta cells; Coxsackie and adenovirus receptor; Coxsackievirus B; Enterovirus; Insulin granule; Pancreas; Protein interacting with C-kinase 1 (PICK1)AdultCoxsackie and Adenovirus Receptor-Like Membrane ProteinAdolescentImmunoprecipitationBlotting WesterninsuliiniArticle03 medical and health sciencesYoung AdultMétabolismeInternal MedicinemedicineHumansImmunoprecipitationPancreasCoxsackie and adenovirus receptorInsulin granuleDiabétologieBeta cellshaima030104 developmental biologyDiabetes Mellitus Type 1Carrier ProteinsDiabetologia
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Phase stability and electronic structure of iridium metal at the megabar range

2019

[EN] The 5d transition metals have attracted specific interest for high-pressure studies due to their extraordinary stability and intriguing electronic properties. In particular, iridium metal has been proposed to exhibit a recently discovered pressure-induced electronic transition, the so-called core-level crossing transition at the lowest pressure among all the 5d transition metals. Here, we report an experimental structural characterization of iridium by x-ray probes sensitive to both long- and short-range order in matter. Synchrotron-based powder x-ray diffraction results highlight a large stability range (up to 1.4 Mbar) of the low-pressure phase. The compressibility behaviour was char…

0301 basic medicineMaterials scienceAbsorption spectroscopySciencechemistry.chemical_elementElectronic structureMolecular electronic transitionArticle03 medical and health sciencessymbols.namesake0302 clinical medicineTransition metalIridiumSpectroscopyCondensed-matter physicsSpectroscopyExafsBulk modulusMultidisciplinaryFermi levelQRCondensed Matter Physics030104 developmental biologychemistryChemical physicsFISICA APLICADAsymbolsMedicineDen kondenserade materiens fysik030217 neurology & neurosurgeryPressures
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The collagen type I segment long spacing (SLS) and fibrillar forms: Formation by ATP and sulphonated diazo dyes.

2016

The collagen type I segment long spacing (SLS) crystallite is a well-ordered rod-like molecular aggregate, ∼300nm in length, which is produced in vitro under mildly acidic conditions (pH 2.5-3.5) in the presence of 1mM ATP. The formation of the SLS crystallite amplifies the inherent linear structural features of individual collagen heterotrimers, due to the punctate linear distribution and summation of the bulkier amino acid side chains along the length of individual collagen heterotrimers. This can be correlated structurally with the 67nm D-banded collagen fibril that is found in vivo, and formed in vitro. Although first described many years ago, the range of conditions required for ATP-in…

0301 basic medicineMaterials sciencePolymersMethyl blueMuscle Fibers SkeletalGeneral Physics and AstronomyFibrilNegative StainingCollagen Type I03 medical and health scienceschemistry.chemical_compoundAdenosine TriphosphateStructural BiologyPolymer chemistryGeneral Materials ScienceColoring AgentsSirius RedEvans Bluechemistry.chemical_classification030102 biochemistry & molecular biologyFibrillogenesisCell BiologyPolyelectrolytesAmino acidCongo redMicroscopy Electron030104 developmental biologychemistryBiophysicsCrystalliteAzo CompoundsEvans BlueMicron (Oxford, England : 1993)
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Great Expectations: Principal Investigator and Trainee Perspectives on Hiring, Supervision, and Mentoring

2018

A functioning mentor–trainee relationship is of high importance in academia. Discrepancies in expectations between principal investigators (PIs) and trainees are a source of misunderstandings and conflicts, endangering scientific progress and career advancement. In this pilot study, we sought to explore the expectations of PIs and trainees, providing consensus data from physician‐scientists and junior researchers who attended an educational workshop, entitled “The EASL/AASLD Masterclass,” in December 2017. Twenty‐three Masterclass attendees, comprising nine trainees (four Ph.D. candidates, five postdoctoral researchers) and 14 PIs, responded to an online survey. Both parties were asked to s…

0301 basic medicineMedical educationHepatologyWork ethicScientific progress4. EducationAcademic developmentMEDLINEMeeting Report3. Good health03 medical and health sciences030104 developmental biology0302 clinical medicinelcsh:Diseases of the digestive system. Gastroenterologylcsh:RC799-869PsychologyCompetence (human resources)030217 neurology & neurosurgeryHepatology Communications
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Towards development of a statistical framework to evaluate myotonic dystrophy type 1 mRNA biomarkers in the context of a clinical trial

2020

AbstractMyotonic dystrophy type 1 (DM1) is a rare genetic disorder, characterised by muscular dystrophy, myotonia, and other symptoms. DM1 is caused by the expansion of a CTG repeat in the 3’-untranslated region of DMPK. Longer CTG expansions are associated with greater symptom severity and earlier age at onset. The primary mechanism of pathogenesis is thought to be mediated by a gain of function of the CUG-containing RNA, that leads to trans-dysregulation of RNA metabolism of many other genes. Specifically, the alternative splicing (AS) and alternative polyadenylation (APA) of many genes is known to be disrupted. In the context of clinical trials of emerging DM1 treatments, it is important…

0301 basic medicineMicroarrayPhysiologyMicroarraysBioinformaticsBiochemistryMachine Learning0302 clinical medicineMathematical and Statistical TechniquesMedicine and Health SciencesMyotonic DystrophyMuscular dystrophyOligonucleotide Array Sequence AnalysisClinical Trials as TopicMultidisciplinaryMusclesQStatisticsRGenetic disorderMuscle AnalysisBody FluidsNucleic acidsBloodBioassays and Physiological AnalysisTreatment OutcomeGenetic DiseasesPhysical SciencesMedicineRegression AnalysisAnatomyDatabases Nucleic AcidResearch Articlemusculoskeletal diseasesGenetic Markerscongenital hereditary and neonatal diseases and abnormalitiesScienceContext (language use)Linear Regression AnalysisBiostatisticsResearch and Analysis MethodsPolyadenylationMyotonic dystrophyMyotonin-Protein Kinase03 medical and health sciencesmedicineGeneticsHumansRNA MessengerStatistical MethodsLeast-Squares AnalysisGeneClinical GeneticsModels Geneticbusiness.industryAlternative splicingBiology and Life Sciencesmedicine.diseaseMyotoniaAlternative Splicing030104 developmental biologyRNA processingRNAGene expressionbusinessTrinucleotide repeat expansionTrinucleotide Repeat Expansion030217 neurology & neurosurgeryBiomarkersMathematicsForecastingPLoS ONE
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Origin of modern syphilis and emergence of a pandemic Treponema pallidum cluster

2016

The abrupt onslaught of the syphilis pandemic that started in the late fifteenth century established this devastating infectious disease as one of the most feared in human history1 . Surprisingly, despite the availability of effective antibiotic treatment since the mid-twentieth century, this bacterial infection, which is caused by Treponema pallidum subsp. pallidum (TPA), has been re-emerging globally in the last few decades with an estimated 10.6 million cases in 2008 (ref. 2). Although resistance to penicillin has not yet been identified, an increasing number of strains fail to respond to the secondline antibiotic azithromycin3. Little is known about the genetic patterns in current infec…

0301 basic medicineMicrobiologia340 LawCiencias de la SaludAzithromycinGlobal HealthBacterisApplied Microbiology and Biotechnology2726 Microbiology (medical)1307 Cell BiologyGenotypePandemicPhylogenyMolecular EpidemiologyTreponemaPhylogenetic treebiology2404 Microbiology10177 Dermatology ClinicTREPONEMA PALLIDUM10218 Institute of Legal MedicineAnti-Bacterial Agents3. Good health590 Animals (Zoology)//purl.org/becyt/ford/3 [https]ORIGIN OF SYPHILISMalalties de transmissió sexualDNA BacterialMicrobiology (medical)CIENCIAS MÉDICAS Y DE LA SALUDGenotypeImmunology610 Medicine & healthMicrobiologyEvolution Molecular//purl.org/becyt/ford/3.3 [https]10127 Institute of Evolutionary Biology and Environmental Studies03 medical and health sciences1311 GeneticsPhylogeneticsDrug Resistance BacterialGeneticsmedicine2402 Applied Microbiology and BiotechnologyHumansSyphilisTreponema pallidumPandemics2403 ImmunologyMolecular epidemiologyGenetic VariationSequence Analysis DNACell Biologymedicine.diseasebiology.organism_classificationVirologyEnfermedades Infecciosas030104 developmental biologyInfectious disease (medical specialty)570 Life sciences; biologySyphilisGenome Bacterial
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Análisis bibliométrico de la producción científica española en Enfermedades Infecciosas y en Microbiología.

2016

Resumen Introduccion El analisis bibliometrico de la produccion y repercusion de los documentos por areas de conocimiento es un indicador cuantitativo y cualitativo de la actividad investigadora realizada en ese campo. El objetivo de este articulo es conocer la contribucion de las instituciones espanolas a la investigacion en Enfermedades Infecciosas y en Microbiologia en los ultimos anos. Material y metodos Se analizaron los documentos publicados en las revistas incluidas en las categorias «Infectious Diseases» y «Microbiology» de la Web of Science (Science Citation Index Expanded) de la ISI Web of Knowledge desde el ano 2000 al 2013. Resultados En Enfermedades Infecciosas, Espana ocupo la…

0301 basic medicineMicrobiology (medical)03 medical and health sciences0302 clinical medicine030106 microbiology030212 general & internal medicineBibliometric analysis Enfermedades infecciosas España Estudio bibliométrico Infectious diseases Microbiology Microbiología Producción científica Scientific production Spain
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