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showing 10 items of 5959 documents

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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Proteomics Reveals the Potential Protective Mechanism of Hydrogen Sulfide on Retinal Ganglion Cells in an Ischemia/Reperfusion Injury Animal Model

2020

Glaucoma is the leading cause of irreversible blindness and is characterized by progressive retinal ganglion cell (RGC) degeneration. Hydrogen sulfide (H2S) is a potent neurotransmitter and has been proven to protect RGCs against glaucomatous injury in vitro and in vivo. This study is to provide an overall insight of H2S&rsquo

0301 basic medicineneuronal apoptosisgenetic structuresQuantitative proteomicshydrogen sulfidePharmaceutical Sciencelcsh:Medicinelcsh:RS1-441PharmacologyProteomicsRetinal ganglionArticlelabel-free mass spectrometrylcsh:Pharmacy and materia medica03 medical and health scienceschemistry.chemical_compound0302 clinical medicinemitochondrial functionIn vivoDrug DiscoverymedicineRetinaChemistrylcsh:RRetinalmedicine.diseaseequipment and supplieseye diseases030104 developmental biologymedicine.anatomical_structureglaucomaRetinal ganglion cellMolecular Medicinesense organsReperfusion injurysignalling pathways030217 neurology & neurosurgeryPharmaceuticals
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Off-Target-Based Design of Selective HIV-1 PROTEASE Inhibitors

2021

The approval of the first HIV-1 protease inhibitors (HIV-1 PRIs) marked a fundamental step in the control of AIDS, and this class of agents still represents the mainstay therapy for this illness. Despite the undisputed benefits, the necessary lifelong treatment led to numerous severe side-effects (metabolic syndrome, hepatotoxicity, diabetes, etc.). The HIV-1 PRIs are capable of interacting with “secondary” targets (off-targets) characterized by different biological activities from that of HIV-1 protease. In this scenario, the in-silico techniques undoubtedly contributed to the design of new small molecules with well-fitting selectivity against the main target, analyzing possible undesirabl…

0301 basic medicineon/off-targetsProtein ConformationComputer sciencemedicine.medical_treatmentHIV InfectionsLigands01 natural sciencesHIV ProteaseHIV-1 proteaseCatalytic DomainDrug DiscoveryBiology (General)DRUDITSpectroscopyMolecular StructurebiologyGeneral MedicineResearch processSmall moleculeComputer Science ApplicationsMolecular Docking SimulationChemistryligand-structure basedQH301-705.5NCI databaseComputational biologyArticleCatalysisInorganic ChemistryStructure-Activity Relationshipmolecular descriptors03 medical and health sciencesHIV-1 proteasemedicineHumansComputer SimulationPhysical and Theoretical ChemistryQD1-999Molecular BiologyVirtual screeningProteaseOrganic ChemistryHIV Protease Inhibitorsmolecular dockingvirtual screening0104 chemical sciences010404 medicinal & biomolecular chemistry030104 developmental biologyDrug DesignHIV-1biology.proteinInternational Journal of Molecular Sciences
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Application of a portable instrument for rapid and reliable detection of SARS-CoV-2 infection in any environment

2020

Abstract The ongoing outbreak of the novel coronavirus (SARS‐CoV‐2) infection is creating serious challenges for health laboratories that seek to identify viral infections as early as possible, optimally at the earliest appearance of symptom. Indeed, there is urgent need to develop and deploy robust diagnostic methodologies not only to use in health laboratory environments but also directly in places where humans circulate and spread the virus such as airports, trains, boats, and any public aggregation places. The success of a reliable and sensitive asymptomatic diagnosis relies on the identification and measurement of informative biomarkers from human host and virus in a rapid, sensitive, …

0301 basic medicineportableSevere acute respiratory syndrome coronavirus 2 (SARS-CoV-2)Pneumonia ViralImmunologycoronavirusBiologymedicine.disease_causeSensitivity and SpecificityDisease OutbreaksWorkflowBetacoronavirus03 medical and health sciencesCOVID-19 Testing0302 clinical medicinePandemicmedicineAnimalsHumansMass ScreeninginstrumentImmunology and AllergyBiomarker discoveryAsymptomatic InfectionsPandemicsdeviceMass screeningCoronavirusInvited ReviewClinical Laboratory TechniquesSARS-CoV-2COVID-19Reproducibility of ResultsClinical Laboratory Servicescoronavirus; device; instrument; portable; SARS-CoV-2 detection; Animals; Asymptomatic Infections; Betacoronavirus; Biomarkers; Clinical Laboratory Services; Clinical Laboratory Techniques; Coronavirus Infections; Disease Outbreaks; Humans; Mass Screening; Pandemics; Pneumonia Viral; Reproducibility of Results; Sensitivity and Specificity; WorkflowIdentification (information)030104 developmental biologyWorkflowRisk analysis (engineering)SARS‐CoV‐2 detectionCoronavirus InfectionsHost (network)Biomarkers030215 immunology
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Tumor Heterogeneity, Single-Cell Sequencing, and Drug Resistance

2016

Tumor heterogeneity has been compared with Darwinian evolution and survival of the fittest. The evolutionary ecosystem of tumors consisting of heterogeneous tumor cell populations represents a considerable challenge to tumor therapy, since all genetically and phenotypically different subpopulations have to be efficiently killed by therapy. Otherwise, even small surviving subpopulations may cause repopulation and refractory tumors. Single-cell sequencing allows for a better understanding of the genomic principles of tumor heterogeneity and represents the basis for more successful tumor treatments. The isolation and sequencing of single tumor cells still represents a considerable technical ch…

0301 basic medicineprecision medicinelcsh:Medicinelcsh:RS1-441Pharmaceutical ScienceReviewsingle-cell sequencingcirculating tumor cellsBiologylaser-capture microdissectionmulti-region sequencingcancer treatmentDNA sequencinglcsh:Pharmacy and materia medicaxenograft tumor models03 medical and health sciencesCirculating tumor cellDrug DiscoveryIllumina dye sequencingMicrodissectionLaser capture microdissectionnext generation sequencingWhole Genome AmplificationGeneticswhole genome amplificationflow cytometrytumor ecosystemslcsh:RRNA sequencing030104 developmental biologySingle cell sequencingintratumoral heterogeneityindividualized therapyMolecular MedicinePyrosequencingmicromanipulationPharmaceuticals
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Antiprotozoal and cysteine proteases inhibitory activity of dipeptidyl enoates

2018

A family of dipeptidyl enoates has been prepared and tested against the parasitic cysteine proteases rhodesain, cruzain and falcipain-2 related to sleeping sickness, Chagas disease and malaria, respectively. They have also been tested against human cathepsins B and L1 for selectivity. Dipeptidyl enoates resulted to be irreversible inhibitors of these enzymes. Some of the members of the family are very potent inhibitors of parasitic cysteine proteases displaying k2nd (M−1s−1) values of seven orders of magnitude. In vivo antiprotozoal testing was also performed. Inhibitors exhibited IC50 values in the micromolar range against Plasmodium falciparum, Trypanosoma brucei, Trypanosoma cruzi and ev…

0301 basic medicinesleeping sicknessClinical BiochemistryPharmaceutical Science01 natural sciencesBiochemistryCathepsin BinhibitorsDrug Discoverychemistry.chemical_classificationbiologyChemistryDipeptidesHep G2 CellsMolecular Docking SimulationCysteine EndopeptidasesBiochemistryAntiprotozoalMolecular MedicineChagas diseaseProteasesCell Survivalmedicine.drug_classPlasmodium falciparumTrypanosoma brucei bruceimalariaAntiprotozoal AgentsCysteine Proteinase InhibitorsTrypanosoma bruceicysteine proteasesInhibitory Concentration 50Structure-Activity Relationship03 medical and health sciencesparasitic diseasesmedicineHumansTrypanosoma cruziMolecular Biologychagas diseaseBinding Sites010405 organic chemistryOrganic ChemistryPlasmodium falciparumbiology.organism_classificationmedicine.diseaseProtein Structure Tertiary0104 chemical sciences030104 developmental biologyEnzymeCysteineBioorganic & Medicinal Chemistry
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In Vivo Cardiotoxicity Induced by Sodium Aescinate in Zebrafish Larvae

2016

Sodium aescinate (SA) is a widely-applied triterpene saponin product derived from horse chestnut seeds, possessing vasoactive and organ-protective activities with oral or injection administration in the clinic. To date, no toxicity or adverse events in SA have been reported, by using routine models (in vivo or in vitro), which are insufficient to predict all aspects of its pharmacological and toxicological actions. In this study, taking advantage of transparent zebrafish larvae (Danio rerio), we evaluated cardiovascular toxicity of SA at doses of 1/10 MNLC, 1/3 MNLC, MNLC and LC10 by yolk sac microinjection. The qualitative and quantitative cardiotoxicity in zebrafish was assessed at 48 h p…

0301 basic medicinesodium aescinateEmbryo NonmammalianHeart malformationDrug Evaluation PreclinicalPharmaceutical Science010501 environmental sciencesPharmacology01 natural sciencesAnalytical ChemistryHeart RateDrug DiscoveryToxicity Tests ChronicZebrafishYolk SacbiologyCommunicationHeartLC10medicine.anatomical_structureChemistry (miscellaneous)LarvaToxicityMolecular MedicineHeart Defects CongenitalMicroinjectionscardiotoxicityHemorrhagelarvaelcsh:QD241-44103 medical and health scienceslcsh:Organic chemistryIn vivoHeart ratemedicineMNLCAnimalsPhysical and Theoretical ChemistryYolk sacAdverse effect0105 earth and related environmental sciencesCardiotoxicityDose-Response Relationship DrugOrganic ChemistryThrombosisSaponinsbiology.organism_classificationzebrafishTriterpenes030104 developmental biologyMolecules
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New Thiazole Nortopsentin Analogues Inhibit Bacterial Biofilm Formation.

2018

New thiazole nortopsentin analogues were conveniently synthesized and evaluated for their activity as inhibitors of biofilm formation of relevant Gram-positive and Gram-negative pathogens. All compounds were able to interfere with the first step of biofilm formation in a dose-dependent manner, showing a selectivity against the staphylococcal strains. The most active derivatives elicited IC50 values against Staphylococcus aureus ATCC 25923, ranging from 0.40&ndash

0301 basic medicinethiazole derivativeAquatic OrganismsIndolesDrug ResistancePharmaceutical ScienceBacterial growthAntibiofilm agentmedicine.disease_cause01 natural scienceschemistry.chemical_compoundDrug Discoveryanti-virulence agents; antibiofilm agents; marine alkaloids; nortopsentin analogues; thiazole derivatives; Anti-Bacterial Agents; Aquatic Organisms; Biofilms; Humans; Imidazoles; Indoles; Inhibitory Concentration 50; Staphylococcal Infections; Staphylococcus aureus; Thiazoles; Drug Resistance; Bacterial; Anti-virulence agents; Antibiofilm agents; Marine alkaloids; Nortopsentin analogues; Thiazole derivativesPharmacology Toxicology and Pharmaceutics (miscellaneous)lcsh:QH301-705.5Aquatic OrganismBiofilmBacterialImidazolesantibiofilm agentsStaphylococcal InfectionsAnti-Bacterial Agentsnortopsentin analoguesBiochemistryStaphylococcus aureusStaphylococcus aureumarine alkaloidsthiazole derivativesSelectivityHumanStaphylococcus aureusAnti-virulence agentNortopsentin analogueArticle03 medical and health sciencesInhibitory Concentration 50Anti-Bacterial AgentDrug Resistance BacterialIc50 valuesmedicineHumansThiazoleImidazoleStaphylococcal Infection010405 organic chemistryBiofilmSettore CHIM/08 - Chimica Farmaceutica0104 chemical sciencesmarine alkaloidThiazoles030104 developmental biologychemistrylcsh:Biology (General)anti-virulence agentsIndoleBiofilmsThiazoleMarine drugs
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Analysis of the Transcriptome of the Red Seaweed Grateloupia imbricata with Emphasis on Reproductive Potential

2018

Grateloupia imbricata is an intertidal marine seaweed and candidate model organism for both industry and academic research, owing to its ability to produce raw materials such as carrageenan. Here we report on the transcriptome of G. imbricata with the aim of providing new insights into the metabolic pathways and other functional pathways related to the reproduction of Grateloupia species. Next-generation sequencing was carried out with subsequent de novo assembly and annotation using state-of-the-art bioinformatic protocols. The results show the presence of transcripts required for the uptake of glycerol, which is a specific carbon source for in vitro culture of G. imbricata and nucleotide …

0301 basic medicineved/biology.organism_classification_rank.speciescarbon sourcesPharmaceutical ScienceRed algaetranscriptome shotgun assemblyreproductionTranscriptome03 medical and health scienceschemistry.chemical_compoundBiosynthesisgrowth regulatorsDrug DiscoveryModel organismlcsh:QH301-705.5Pharmacology Toxicology and Pharmaceutics (miscellaneous)red algaeMethyl jasmonatebiologyved/biologybiology.organism_classificationSporeMetabolic pathway030104 developmental biologylcsh:Biology (General)chemistryBiochemistryPolyamineMarine Drugs
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Synthesis and cytotoxic activity of new artemisinin hybrid molecules against human leukemia cells

2017

A series of new artemisinin-derived hybrids which incorporate cholic acid moieties have been synthesized and evaluated for their antileukemic activity against sensitive CCRF-CEM and multidrug-resistant CEM/ADR5000 cells. The new hybrids 20-28 showed IC50 values in the range of 0.019µM-0.192µM against CCRF-CEM cells and between 0.345µM and 7.159µM against CEM/ADR5000 cells. Amide hybrid 25 proved the most active compound against both CCRF-CEM and CEM/ADR5000 cells with IC50 value of 0.019±0.001µM and 0.345±0.031µM, respectively. A relatively low cross resistance to hybrids 20-28 in the range of 5.7-fold to 46.1-fold was measured. CEM/ADR5000 cells showed higher resistance than CCRF-CEM to al…

0301 basic medicinevirusesClinical BiochemistryPharmaceutical ScienceAntineoplastic AgentsBiochemistryAntileukemic agentStructure-Activity Relationship03 medical and health scienceschemistry.chemical_compound0302 clinical medicineimmune system diseaseshemic and lymphatic diseasesAmideDrug DiscoveryTumor Cells CulturedmedicineHumansCytotoxic T cellDoxorubicinArtemisininMolecular BiologyIC50Cross-resistanceCell ProliferationLeukemiaDose-Response Relationship DrugMolecular StructureOrganic ChemistryCholic acidhemic and immune systemsArtemisinins030104 developmental biologyBiochemistrychemistry030220 oncology & carcinogenesisMolecular MedicineDrug Screening Assays Antitumormedicine.drugBioorganic & Medicinal Chemistry
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