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showing 10 items of 19256 documents

Synthesis and antiproliferative mechanism of action of pyrrolo[3′,2′:6,7] cyclohepta[1,2-d]pyrimidin-2-amines as singlet oxygen photosensitizers

2016

A new series of pyrrolo[3′,2′:6,7]cyclohepta[1,2-d]pyrimidin-2-amines, was conveniently prepared using a versatile and high yielding multistep sequence. A good number of derivatives was obtained and the cellular photocytotoxicity was evaluated in vitro against three different human tumor cell lines with EC50 (0.08–4.96 μM) values reaching the nanomolar level. Selected compounds were investigated by laser flash photolysis. The most photocytotoxic derivative, exhibiting a fairly long-lived triplet state (τ ∼ 7 μs) and absorbance in the UV–Vis, was tested in the photo-oxidations of 9,10-anthracenedipropionic acid (ADPA) by singlet oxygen. The photosentizing properties are responsible for the c…

0301 basic medicinemedicine.medical_treatmentPhotodynamic therapyChemistry Techniques SyntheticAntiproliferative activityPhotochemistry01 natural sciencesPhotodynamic therapychemistry.chemical_compound7]cyclohepta[1Drug DiscoveryTriplet stateAmineschemistry.chemical_classificationPhotosensitizing AgentsCell DeathSinglet OxygenChemistrySinglet oxygenGeneral MedicineAntiproliferative activity; Photodynamic therapy; Photosensitizing agents; Pyrrolo[3′; 2′:6; 7]cyclohepta[1; 2-d]pyrimidin-2-amines; Reactive oxygen species; Drug Discovery3003 Pharmaceutical Science; Organic Chemistry; PharmacologyPyrrolo[3′2′:67]cyclohepta[12-d]pyrimidin-2-amineSettore ING-IND/22 - Scienza E Tecnologia Dei Materiali2-d]pyrimidin-2-aminesFlash photolysisReactive oxygen specieKineticsAntineoplastic AgentsAbsorbance03 medical and health sciencesCell Line TumormedicineHumansPyrrolo[3′Cell ProliferationPharmacologyReactive oxygen speciesPhotosensitizing agentPhotolysis010405 organic chemistry2′:6Drug Discovery3003 Pharmaceutical SciencePhotodissociationOrganic ChemistryCombinatorial chemistrySettore CHIM/08 - Chimica Farmaceutica0104 chemical sciencesAntiproliferative activity; Photodynamic therapy; Photosensitizing agents; Pyrrolo[3′2′:67]cyclohepta[12-d]pyrimidin-2-amines; Reactive oxygen species; Drug Discovery3003 Pharmaceutical Science; Organic Chemistry; PharmacologyKinetics030104 developmental biologyDrug DesignReactive oxygen species
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Immunogenicity of a Fully Synthetic MUC1 Glycopeptide Antitumor Vaccine Enhanced by Poly(I:C) as a TLR3-Activating Adjuvant.

2017

Fully synthetic MUC1 glycopeptide antitumor vaccines have a precisely specified structure and induce a targeted immune response without suppression of the immune response when using an immunogenic carrier protein. However, tumor-associated aberrantly glycosylated MUC1 glycopeptides are endogenous structures, “self-antigens”, that exhibit only low immunogenicity. To overcome this obstacle, a fully synthetic MUC1 glycopeptide antitumor vaccine was combined with poly(inosinic acid:cytidylic acid), poly(I:C), as a structurally defined Toll-like receptor 3 (TLR3)-activating adjuvant. This vaccine preparation elicited extraordinary titers of IgG antibodies which strongly bound human breast cancer…

0301 basic medicinemedicine.medical_treatmentchemical and pharmacologic phenomenaBiochemistryCancer Vaccines03 medical and health sciencesMice0302 clinical medicineImmune systemCancer immunotherapyAdjuvants ImmunologicDrug DiscoverymedicineAnimalsHumansGeneral Pharmacology Toxicology and PharmaceuticsMUC1PharmacologyVaccines SyntheticbiologyChemistryImmunogenicityOrganic ChemistryMucin-1GlycopeptidesDendritic CellsVirologyGlycopeptideToll-Like Receptor 3030104 developmental biologyPoly I-C030220 oncology & carcinogenesisTLR3biology.proteinMolecular MedicineAntibodyAdjuvantChemMedChem
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Drosophila Food-Associated Pheromones: Effect of Experience, Genotype and Antibiotics on Larval Behavior

2016

International audience; Animals ubiquitously use chemical signals to communicate many aspects of their social life. These chemical signals often consist of environmental cues mixed with species-specific signals-pheromones-emitted by conspecifics. During their life, insects can use pheromones to aggregate, disperse, choose a mate, or find the most suitable food source on which to lay eggs. Before pupariation, larvae of several Drosophila species migrate to food sources depending on their composition and the presence of pheromones. Some pheromones derive from microbiota gut activity and these food-associated cues can enhance larval attraction or repulsion. To explore the mechanisms underlying…

0301 basic medicinemelanogasterlcsh:Medicine[ SDV.BA ] Life Sciences [q-bio]/Animal biologyBiochemistryPheromonesLarvaeAntibioticsMedicine and Health Sciencesinsectslcsh:ScienceAnimal Signaling and CommunicationLarvaMultidisciplinaryInsect MetamorphosisbiologyAnimal BehaviorBehavior AnimalEcologyAntimicrobialscommunicationDrosophila Melanogaster[SDV.BA]Life Sciences [q-bio]/Animal biologyaggressionsex-pheromonesDrugsAnimal ModelsAttractionPupaSex pheromoneLarvacourtshipNeurosciences (Sciences cognitives)DrosophilaDrosophila melanogasterCuesrecognitionPupariationResearch ArticleattractionComputer and Information SciencesArthropodaGenotypeZoologyResearch and Analysis MethodsMicrobiology03 medical and health sciencesModel OrganismsInvertebrate ZoologySEX-PHEROMONES;MELANOGASTER;AGGRESSION;COURTSHIP;COMMUNICATION;RECOGNITION;ATTRACTION;EVOLUTION;MUTATION;INSECTSMicrobial ControlevolutionAnimalsDrosophilaSensory cuePharmacologyBehaviorMetamorphosisData Visualizationlcsh:RfungiOrganismsBiology and Life SciencesPupaebiology.organism_classificationZoologie des invertébrésInvertebratesColor Codes030104 developmental biologyFoodOdorantslcsh:QmutationZoologyEntomologyNeuroscienceDevelopmental Biology
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Using Zebrafish to Model Autism Spectrum Disorder: A Comparison of ASD Risk Genes Between Zebrafish and Their Mammalian Counterparts.

2020

Autism spectrum disorders (ASDs) are a highly variable and complex set of neurological disorders that alter neurodevelopment and cognitive function, which usually presents with social and learning impairments accompanied with other comorbid symptoms like hypersensitivity or hyposensitivity, or repetitive behaviors. Autism can be caused by genetic and/or environmental factors and unraveling the etiology of ASD has proven challenging, especially given that different genetic mutations can cause both similar and different phenotypes that all fall within the autism spectrum. Furthermore, the list of ASD risk genes is ever increasing making it difficult to synthesize a common theme. The use of ro…

0301 basic medicinemiceautismNeurosciences. Biological psychiatry. NeuropsychiatryDiseaseReviewbehavioral disciplines and activitiesASD03 medical and health sciencesCellular and Molecular Neuroscience0302 clinical medicinemental disordersmedicinehumangenesMolecular BiologyZebrafishGenebiologybehaviorCorrectionCognitionmedicine.diseasebiology.organism_classificationzebrafishPhenotype030104 developmental biologyAutism spectrum disorderAutismMolecular NeuroscienceNeuroscience030217 neurology & neurosurgeryNeuroscienceRC321-571Frontiers in Molecular Neuroscience
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Efficient and reproducible experimental infections of rats with Blastocystis spp.

2018

Although Blastocystis spp. infect probably more than 1 billion people worldwide, their clinical significance is still controversial and their pathophysiology remains poorly understood. In this study, we describe a protocol for an efficient and reproducible model of chronic infection in rats, laying the groundwork for future work to evaluate the pathogenic potential of this parasite. In our experimental conditions, we were unable to infect rats using vacuolar forms of an axenically cultivated ST4 isolate, but we successfully established chronic infections of 4 week-old rats after oral administration of both ST3 and ST4 purified cysts isolated from human stool samples. The infection protocol …

0301 basic medicinemodèle animal[SDV]Life Sciences [q-bio]lcsh:MedicineBlastocystis Infections[SDV.BC.IC] Life Sciences [q-bio]/Cellular Biology/Cell Behavior [q-bio.CB]souris[SDV.IMM.II]Life Sciences [q-bio]/Immunology/Innate immunityFecesblastocyste[SDV.BC.IC]Life Sciences [q-bio]/Cellular Biology/Cell Behavior [q-bio.CB]Medicine and Health SciencesParasite hostingCystratmodèle pour les maladies humaineslcsh:Scienceblastocyst stageProtozoansGastrointestinal tractMice Inbred BALB CMice Inbred C3HMultidisciplinarybiologyaxenic cultureEukaryotaPathophysiologyanimal models3. Good health[SDV] Life Sciences [q-bio]Separation ProcessesExperimental Organism SystemsAnatomyResearch ArticlemiceColonMouse ModelsResearch and Analysis MethodsMicrobiologyculture axeniqueMicrobiology03 medical and health sciencesModel OrganismsmedicineParasitic DiseasesAnimalsHumansClinical significanceAnimal Models of Disease[SDV.IMM.II] Life Sciences [q-bio]/Immunology/Innate immunityDistillationBlastocystisHost (biology)lcsh:ROrganismsBiology and Life Sciencesbiology.organism_classificationmedicine.disease[SDV.MP.BAC]Life Sciences [q-bio]/Microbiology and Parasitology/BacteriologyParasitic ProtozoansRatsMice Inbred C57BLGastrointestinal TractChronic infectionDisease Models AnimalAnimal Models of Infection030104 developmental biologyBlastocystisAnimal Studieslcsh:Q[SDV.MP.BAC] Life Sciences [q-bio]/Microbiology and Parasitology/BacteriologyParasitic Intestinal DiseasesDigestive System
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Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models

2019

Myotonic dystrophy type 1 (DM1) is a life-threatening and chronically debilitating neuromuscular disease caused by the expansion of a CTG trinucleotide repeat in the 3′ UTR of the DMPK gene. The mutant RNA forms insoluble structures capable of sequestering RNA binding proteins of the Muscleblind-like (MBNL) family, which ultimately leads to phenotypes. In this work, we demonstrate that treatment with the antiautophagic drug chloroquine was sufficient to up-regulate MBNL1 and 2 proteins in Drosophila and mouse (HSA LR ) models and patient-derived myoblasts. Extra Muscleblind was functional at the molecular level and improved splicing events regulated by MBNLs in all disease models. In vivo,…

0301 basic medicinemusculoskeletal diseasesMaleRNA SplicingRNA-binding proteinBiologyMyotonic dystrophychloroquinemuscleblindMyoblasts03 medical and health scienceschemistry.chemical_compoundMice0302 clinical medicineIn vivomedicineAutophagyMBNL1AnimalsDrosophila ProteinsHumansMyotonic DystrophytherapyMultidisciplinarymyotonic dystrophyMusclesRNANuclear ProteinsRNA-Binding ProteinsChloroquinemedicine.diseaseMyotoniaCell biologyDNA-Binding ProteinsDisease Models Animal030104 developmental biologyPhenotypechemistryPNAS PlusRNA splicingDrosophilaFemaleTrinucleotide repeat expansion030217 neurology & neurosurgery
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miR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models

2018

Functional depletion of the alternative splicing factors Muscleblind-like (MBNL 1 and 2) is at the basis of the neuromuscular disease myotonic dystrophy type 1 (DM1). We previously showed the efficacy of miRNA downregulation in Drosophila DM1 model. Here, we screen for miRNAs that regulate MBNL1 and MBNL2 in HeLa cells. We thus identify miR-23b and miR-218, and confirm that they downregulate MBNL proteins in this cell line. Antagonists of miR-23b and miR-218 miRNAs enhance MBNL protein levels and rescue pathogenic missplicing events in DM1 myoblasts. Systemic delivery of these “antagomiRs” similarly boost MBNL expression and improve DM1-like phenotypes, including splicing alterations, histo…

0301 basic medicinemusculoskeletal diseasesMalecongenital hereditary and neonatal diseases and abnormalitiesScienceMyoblasts SkeletalGeneral Physics and AstronomyMice TransgenicBiologyMyotonic dystrophyGeneral Biochemistry Genetics and Molecular BiologyArticleCell Line03 medical and health scienceschemistry.chemical_compoundMice0302 clinical medicineRNA interferencemicroRNAmedicineMBNL1Gene silencingAnimalsHumansMyotonic DystrophyGene SilencingRNA Messengerlcsh:ScienceMuscle Skeletal3' Untranslated RegionsMultidisciplinaryThree prime untranslated regionAlternative splicingQRNA-Binding ProteinsGeneral Chemistrymedicine.diseaseMyotoniaCell biologyUp-RegulationAlternative SplicingDisease Models AnimalMicroRNAs030104 developmental biologyPhenotypechemistrylcsh:Q030217 neurology & neurosurgeryHeLa CellsNature Communications
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Association between ribs shape and pulmonary function in patients with Osteogenesis Imperfecta

2019

Graphical abstract

0301 basic medicinemusculoskeletal diseasesVital capacityPulmonary functionMedicinaKyphosisScoliosisPulmonary function testing03 medical and health sciencesFEV1/FVC ratio0302 clinical medicinemedicineRespiratory functionLung volumeslcsh:Science (General)ComputingMethodologies_COMPUTERGRAPHICSRib cagelcsh:R5-920Geometric morphometricsMultidisciplinarybusiness.industryAnatomymedicine.diseasemusculoskeletal system030104 developmental biologyScoliosis030220 oncology & carcinogenesisRib cageOriginal ArticleOsteogenesis imperfectaThoracic spinebusinesslcsh:Medicine (General)lcsh:Q1-390Journal of Advanced Research 21: 177-185 (2020)
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Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

2020

Myotonic dystrophy type 1 (DM1) is a chronically debilitating, rare genetic disease that originates from an expansion of a noncoding CTG repeat in the dystrophia myotonica protein kinase (DMPK) gene. The expansion becomes pathogenic when DMPK transcripts contain 50 or more repetitions due to the sequestration of the muscleblind-like (MBNL) family of proteins. Depletion of MBNLs causes alterations in splicing patterns in transcripts that contribute to clinical symptoms such as myotonia and muscle weakness and wasting. We previously found that microRNA (miR)-23b directly regulates MBNL1 in DM1 myoblasts and mice and that antisense technology (“antagomiRs”) blocking this microRNA (miRNA) boost…

0301 basic medicinemusculoskeletal diseasescongenital hereditary and neonatal diseases and abnormalitiesMyotonic dystrophyArticleantagomiR03 medical and health scienceschemistry.chemical_compound0302 clinical medicineDrug DiscoverymicroRNAMedicineMBNL1AntagomirProtein kinase AmiRNAmyotonic dystrophybusiness.industrylcsh:RM1-950Muscle weaknessmedicine.diseaseMyotoniaMbnl1030104 developmental biologylcsh:Therapeutics. Pharmacologychemistry030220 oncology & carcinogenesisRNA splicingCancer researchHSALR miceMolecular Medicinemedicine.symptomDM1antisense oligonucleotidesbusinessMolecular Therapy: Nucleic Acids
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Ferritin and C-reactive protein are predictive biomarkers of mortality and macrophage activation syndrome in adult onset Still's disease. Analysis of…

2020

ObjectiveTo assess the predictive role of ferritin and C-reactive protein (CRP) on occurrence of macrophage activation syndrome (MAS) and mortality in patients with adult onset Still's disease (AOSD), a rare and severe disease, included in the multicentre Gruppo Italiano di Ricerca in Reumatologia Clinica e Sperimentale (GIRRCS) cohort.MethodsThe predictive role, at the time of diagnosis, of serum levels of ferritin and CRP on occurrence of MAS and mortality, was evaluated by logistic regression analyses and receiver-operating characteristic (ROC) curves were built to identify patients at high risk of MAS and mortality, respectively.ResultsIn assessed 147 patients with AOSD, levels of ferri…

0301 basic medicinemyalgiaAdult-OnsetMalePredictive Value of TestPathology and Laboratory MedicineLogistic regressionBiochemistryGastroenterologyCohort StudiesMathematical and Statistical Techniques0302 clinical medicineMedicine and Health SciencesMultidisciplinarybiologyMacrophage Activation SyndromeStatisticsQRMiddle AgedC-Reactive ProteinsC-Reactive ProteinPredictive value of testsPhysical SciencesCohortRegression AnalysisMedicineFemalemedicine.symptomStill's Disease Adult-OnsetResearch ArticleCohort studyHumanAdultmedicine.medical_specialtyPatientsLogistic ModelInflammatory DiseasesSciencePainStill's Disease Adult-Onset.Research and Analysis Methods03 medical and health sciencesAdult; Biomarkers; C-Reactive Protein; Cohort Studies; Female; Ferritins; Humans; Logistic Models; Macrophage Activation Syndrome; Male; Middle Aged; Predictive Value of Tests; ROC Curve; Still's Disease Adult-OnsetSigns and SymptomsDiagnostic MedicinePredictive Value of TestsInternal medicinemedicineHumansStatistical Methods030203 arthritis & rheumatologyFerritinbusiness.industryC-reactive proteinBiology and Life SciencesProteinsProtein ComplexesMyalgiaBiomarkermedicine.diseaseStill's DiseaseHealth CareFerritin030104 developmental biologyLogistic ModelsROC CurveMacrophage activation syndromeMultivariate AnalysisFerritinsbiology.proteinCohort StudiebusinessMathematicsBiomarkers
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