2023
Novel epigenetic molecular therapies for imprinting disorders
Wang S, Jiang Y. Novel epigenetic molecular therapies for imprinting disorders. Molecular Psychiatry 2023, 28: 3182-3193. PMID: 37626134, PMCID: PMC10618104, DOI: 10.1038/s41380-023-02208-7.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsActive alleleImprinting disordersMolecular mechanismsGenome editing approachesEpigenetic-based therapiesUnique molecular mechanismGenomic imprinting disordersImprinted genesGenome editingMolecular approachesEditing approachesInactive allelesNew therapeutic strategiesAllelesSmall moleculesMolecular therapyTherapeutic strategies
2020
Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy
Cohen J, DeSimone A, Lek M, Lek A. Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy. Trends In Molecular Medicine 2020, 27: 123-137. PMID: 33092966, PMCID: PMC8048701, DOI: 10.1016/j.molmed.2020.09.008.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAnimalsBiomarkersClinical Studies as TopicCombined Modality TherapyDisease ManagementDisease SusceptibilityDrug DevelopmentDrug Evaluation, PreclinicalGenetic Predisposition to DiseaseHomeodomain ProteinsHumansMolecular Targeted TherapyMuscular Dystrophy, FacioscapulohumeralOxidation-ReductionTreatment OutcomeConceptsFacioscapulohumeral muscular dystrophyMuscular dystrophyClinical outcome measuresClinical trialsOutcome measuresTherapeutic approachesMost trialsMolecular therapyEarly trialsTherapeutic developmentTrialsCommon typeCausal roleDystrophyTherapeutic discoveryFSHD researchCombined advancesTherapyGlioblastoma in adults: a Society for Neuro-Oncology (SNO) and European Society of Neuro-Oncology (EANO) consensus review on current management and future directions
Wen PY, Weller M, Lee EQ, Alexander BM, Barnholtz-Sloan JS, Barthel FP, Batchelor TT, Bindra RS, Chang SM, Chiocca EA, Cloughesy TF, DeGroot JF, Galanis E, Gilbert MR, Hegi ME, Horbinski C, Huang RY, Lassman AB, Le Rhun E, Lim M, Mehta MP, Mellinghoff IK, Minniti G, Nathanson D, Platten M, Preusser M, Roth P, Sanson M, Schiff D, Short SC, Taphoorn MJB, Tonn JC, Tsang J, Verhaak RGW, von Deimling A, Wick W, Zadeh G, Reardon DA, Aldape KD, van den Bent MJ. Glioblastoma in adults: a Society for Neuro-Oncology (SNO) and European Society of Neuro-Oncology (EANO) consensus review on current management and future directions. Neuro-Oncology 2020, 22: 1073-1113. PMID: 32328653, PMCID: PMC7594557, DOI: 10.1093/neuonc/noaa106.Peer-Reviewed Original ResearchConceptsNeuro-oncologyConsensus reviewCurrent managementMalignant primary brain tumorIsocitrate dehydrogenase-wildtype glioblastomaPrimary brain tumorsNovel therapiesViral therapyBrain tumorsImportant causeMolecular pathogenesisMolecular therapyEuropean AssociationTherapyEuropean SocietyCommon formGlioblastomaWildtype glioblastomaTumorsFuture directionsImportant advancesImmunotherapyMorbidityPatientsDNA damage responseChapter 6 Molecular genetics of meningiomas
Youngblood MW, Günel M. Chapter 6 Molecular genetics of meningiomas. Handbook Of Clinical Neurology 2020, 169: 101-119. PMID: 32553282, DOI: 10.1016/b978-0-12-804280-9.00006-8.Peer-Reviewed Original ResearchConceptsMolecular mechanismsNext-generation sequencing techniquesComprehensive genomic studiesRecent genomic findingsGenomic studiesPotential molecular therapyMolecular geneticsSequencing techniquesGenomic pathwayOncogenic pathwaysNumber eventsGenomic findingsDriver mutationsMutationsBasic science investigatorsPathwayMolecular therapyImportant insightsEmbryological originCommon brain tumorMolecular subgroupsGeneticsTumorigenesisMechanismProgression
2019
Review of the Current Basic Science Strategies to Treat Critical Limb Ischemia
Haghighat L, Ionescu CN, Regan CJ, Altin SE, Attaran RR, Mena-Hurtado CI. Review of the Current Basic Science Strategies to Treat Critical Limb Ischemia. Vascular And Endovascular Surgery 2019, 53: 316-324. PMID: 30808262, DOI: 10.1177/1538574419831489.Peer-Reviewed Original ResearchConceptsCritical limb ischemiaLimb ischemiaDevelopment of CLIMolecular therapyPoor surgical candidatesImpairment of angiogenesisSurgical candidatesMicrovascular changesNovel imaging modalityTreatment optionsMorbid diseaseTranslational medicine studiesTherapyMedicine studiesImaging modalitiesIschemiaDiseasePatientsPathophysiologyArteriogenesisImpairmentTrials
2017
Emerging concepts and recent advances in basal cell carcinoma
Totonchy M, Leffell D. Emerging concepts and recent advances in basal cell carcinoma. F1000Research 2017, 6: 2085. PMID: 29259776, PMCID: PMC5717469, DOI: 10.12688/f1000research.11314.1.Peer-Reviewed Original ResearchBasal cell carcinomaTreatment of BCCCell carcinomaIncidence of BCCNon-keratinizing cellsHigh cure ratesNon-surgical interventionsNon-surgical approachStandard of careMohs micrographic surgeryTargeted molecular therapiesRecurrent diseaseSurgical excisionCommon malignancySurgical interventionCure rateMicrographic surgeryMolecular therapyCarcinomaBasal layerTreatmentBurden of managementInterventionMorbiditySurgery
2012
A Cell-Penetrating Bispecific Antibody for Therapeutic Regulation of Intracellular Targets
Weisbart RH, Gera JF, Chan G, Hansen JE, Li E, Cloninger C, Levine AJ, Nishimura RN. A Cell-Penetrating Bispecific Antibody for Therapeutic Regulation of Intracellular Targets. Molecular Cancer Therapeutics 2012, 11: 2169-2173. PMID: 22863609, DOI: 10.1158/1535-7163.mct-12-0476-t.Peer-Reviewed Original ResearchConceptsBispecific antibodiesBispecific single-chain Fv fragmentsMonoclonal antibody 3G5Cancer therapyDelivery of antibodiesIntracellular targetsMolecular therapyTherapeutic useTherapeutic regulationMonoclonal antibodiesTherapyAntibodiesMAb 3E10Single-chain Fv fragmentInhibits growthP53 levelsImportant targetMDM2Limited accessFv fragmentTargetTumors
2008
IMPACT (Imaging and Molecular Markers for Patients with Lung Cancer: Approaches with Molecular Targets and Complementary, Innovative and Therapeutic Modalities)
Hong W, Herbst R. IMPACT (Imaging and Molecular Markers for Patients with Lung Cancer: Approaches with Molecular Targets and Complementary, Innovative and Therapeutic Modalities). 2008 DOI: 10.21236/ada483184.Peer-Reviewed Original ResearchMalignant pleural effusionRaf/MEK/ERK pathwayMEK/ERK pathwayRAS/RAF/MEK/ERK pathwayAdvanced diseasePleural effusionLung cancerNSCLC pathogenesisMolecular therapyEGFR pathwayERK pathwayCD-ROM programSignal transduction pathwaysImaging techniquesPathwayTransduction pathwaysPeptide approachPulmonologistsEffusionPathogenesisTherapyCancerDiseasePreventionReceptorsIMPACT (Imaging and Molecular Markers for Patients with Lung Cancer: Approaches with Molecular Targets and Complementary, Innovative and Therapeutic Modalities)
Hong W, Herbst R. IMPACT (Imaging and Molecular Markers for Patients with Lung Cancer: Approaches with Molecular Targets and Complementary, Innovative and Therapeutic Modalities). 2008 DOI: 10.21236/ada486510.Peer-Reviewed Original ResearchMalignant pleural effusionRaf/MEK/ERK pathwayMEK/ERK pathwayRAS/RAF/MEK/ERK pathwayAdvanced diseasePleural effusionLung cancerNSCLC pathogenesisMolecular therapyEGFR pathwayERK pathwayCD-ROM programSignal transduction pathwaysImaging techniquesPathwayTransduction pathwaysPulmonologistsEffusionPathogenesisTherapyCancerDiseasePreventionReceptors
2007
Intranuclear Protein Transduction through a Nucleoside Salvage Pathway*
Hansen JE, Tse CM, Chan G, Heinze ER, Nishimura RN, Weisbart RH. Intranuclear Protein Transduction through a Nucleoside Salvage Pathway*. Journal Of Biological Chemistry 2007, 282: 20790-20793. PMID: 17525162, DOI: 10.1074/jbc.c700090200.Peer-Reviewed Original ResearchConceptsNucleoside salvage pathwaySalvage pathwaySpecific nuclear localizationCargo proteinsGene regulationMolecular delivery vehiclesProtein transportProtein transductionTranscription factorsNuclear localizationGene expressionNuclear membraneHuman diseasesLiving cellsCell nucleiCell membraneNew molecular therapiesNumerous diseasesPathwayTransductionCellsRegulationMolecular therapyFirst demonstrationNew avenues
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