2025
Challenges in Gaucher disease: Perspectives from an expert panel
Grabowski G, Kishnani P, Alcalay R, Prakalapakorn S, Rosenbloom B, Tuason D, Weinreb N. Challenges in Gaucher disease: Perspectives from an expert panel. Molecular Genetics And Metabolism 2025, 145: 109074. PMID: 40112481, DOI: 10.1016/j.ymgme.2025.109074.Peer-Reviewed Original ResearchGD type 1Enzyme replacement therapyNeuronopathic diseaseGD type 2Type 1Central nervous systemMagnetic resonance imagingGaucher diseaseDixon quantitative chemical shift imagingFirst-line treatment of adultsEnzyme replacement therapy productsDual-energy X-ray absorptiometry scanPrompt initiation of treatmentEffects of enzyme replacement therapyX-ray absorptiometry scanQuantitative chemical shift imagingSymptoms of central nervous systemGD type 1 patientsGD type 3Non-Hodgkin's lymphomaPeak bone massFirst-line treatmentType 2Nervous systemInitiation of treatmentA positron emission tomography tracer for the imaging of oxidative stress in the central nervous system
Wilde J, Sun Y, Simpson S, Hill E, Fu Z, Bian E, Kinkaid M, Villanueva P, Weybright A, Terrell W, Qureshi Z, Perera S, Sheppard H, Stone J, Kundu B, Kuan C, Neumann K. A positron emission tomography tracer for the imaging of oxidative stress in the central nervous system. Nature Biomedical Engineering 2025, 1-14. PMID: 40044816, DOI: 10.1038/s41551-025-01362-3.Peer-Reviewed Original ResearchCentral nervous systemPositron emission tomographyInjection of sodium nitroprussideOxidative stressNervous systemClinical trials of antioxidantsMiddle cerebral arteryBlood-brain barrierTrials of antioxidantsPositron emission tomography tracersIntrastriatal injectionParametric mappingSodium nitroprussideCerebral arteryLongitudinally in vivoIn vivo quantificationQuantification of oxidative stressEmission tomographyPET imagingAntioxidant edaravoneDynamic PET imagesHuman plasmaLongitudinal CNS and systemic T-lymphocyte and monocyte activation before and after antiretroviral therapy beginning in primary HIV infection
Chan P, Li X, Li F, Emu B, Price R, Spudich S. Longitudinal CNS and systemic T-lymphocyte and monocyte activation before and after antiretroviral therapy beginning in primary HIV infection. Frontiers In Immunology 2025, 16: 1531828. PMID: 40070827, PMCID: PMC11893981, DOI: 10.3389/fimmu.2025.1531828.Peer-Reviewed Original ResearchConceptsCerebrospinal fluid HIV RNAPrimary HIV infectionT cell activationCD8+ T cell activationAntiretroviral therapyHIV RNACD4+HIV infectionCerebrospinal fluidT cellsMonocyte activationNeuronal injuryCD4+ T cell activationCD8<sup>+</sup> T cellsCD8+ T cellsFrequencies of activated CD4+ART-naive peopleChronic HIV infectionTrafficking of immune cellsAntiretroviral therapy initiationChemokine receptor expressionMultiparameter flow cytometryImmune cell activationSurface marker expressionCentral nervous systemPET Mapping of Receptor Occupancy Using Joint Direct Parametric Reconstruction
Marin T, Belov V, Chemli Y, Ouyang J, Najmaoui Y, Fakhri G, Duvvuri S, Iredale P, Guehl N, Normandin M, Petibon Y. PET Mapping of Receptor Occupancy Using Joint Direct Parametric Reconstruction. IEEE Transactions On Biomedical Engineering 2025, 72: 1057-1066. PMID: 39446540, PMCID: PMC11875991, DOI: 10.1109/tbme.2024.3486191.Peer-Reviewed Original ResearchCentral nervous systemReceptor occupancyLow-binding regionsPET scansSimulation resultsPreclinical in vivo experimentsDynamic PET scansPairs of baselineEstimation of receptor occupancyEstimation frameworkPET neuroimagingReconstruction frameworkModulating drugsTime activity curvesParametric reconstructionDevelopment of drugsLong-Term Engraftment of Cryopreserved Human Neurons for In Vivo Disease Modeling in Neurodegenerative Disease
Marmion D, Deng P, Hiller B, Lewis R, Harms L, Cameron D, Nolta J, Kordower J, Fink K, Wakeman D. Long-Term Engraftment of Cryopreserved Human Neurons for In Vivo Disease Modeling in Neurodegenerative Disease. Biology 2025, 14: 217. PMID: 40001985, PMCID: PMC11852092, DOI: 10.3390/biology14020217.Peer-Reviewed Original ResearchCentral nervous systemStriatum of Sprague-Dawley ratsForebrain GABAergic neuronsLong-term engraftmentHuman induced pluripotent stem cellsHuman neuronsSprague-Dawley ratsWild-type controlsPluripotent stem cellsPrefrontal cortexMature neuronal phenotypeRNU ratsHost striatumDisease in vivoGABAergic neuronsPost-transplantationImmunodeficient ratsIn vivo disease modelsIGABAHuntington's diseaseCynomolgus monkeysNeuronal phenotypeHD miceCNS environmentNeuronal survivalMultiple sclerosis diagnosis and its differential diagnosis in patients presenting with type four ‘mirror pattern’ CSF oligoclonal bands
Marastoni D, Sicchieri M, Pizzini F, Scartezzini A, Virla F, Turano E, Anni D, Bertolazzo M, Ziccardi S, Camera V, Tamanti A, Marini M, Lippi G, Bonetti B, Solomon A, Calabrese M. Multiple sclerosis diagnosis and its differential diagnosis in patients presenting with type four ‘mirror pattern’ CSF oligoclonal bands. Journal Of Neurology 2025, 272: 207. PMID: 39954124, PMCID: PMC11829897, DOI: 10.1007/s00415-025-12947-y.Peer-Reviewed Original ResearchConceptsCentral nervous systemCSF oligoclonal bandsOligoclonal bandsDiagnosis of MSAlternative diagnosesCerebrospinal fluidDemyelinating eventCentral nervous system demyelinating eventMultiple sclerosisInitial central nervous systemOligoclonal banding patternsRadiological follow-upDiagnostic work-upFrequent alternative diagnosesGroup of patientsMethodsSeventy-six patientsCentral nervous system disordersConclusionThe diagnosisMultiple sclerosis diagnosisClinical attacksMRI lesionsParaclinical examinationsDifferential diagnosisFollow-upWork-upComparative analysis of AAV serotypes for transduction of olfactory sensory neurons
Belfort B, Jia J, Garza A, Insalaco A, McGinnis J, Pekarek B, Ortiz-Guzman J, Tepe B, Chen H, Investigators A, Liu Z, Arenkiel B, Arenkiel B, Liu Z, Mollenhauer B, Penninger J, Rousseaux M, Saghatelyan A, Salmaso N, Stadelmann C, Schlossmacher M, Tomlinson J, Woulfe J, Wang Y, Chen Z, Jeong M. Comparative analysis of AAV serotypes for transduction of olfactory sensory neurons. Frontiers In Neuroscience 2025, 19: 1531122. PMID: 40018362, PMCID: PMC11865030, DOI: 10.3389/fnins.2025.1531122.Peer-Reviewed Original ResearchOlfactory sensory neuronsAAV serotypesSensory neuronsCentral nervous systemOptimal AAV serotypeAdeno-associated virusNervous systemGene therapyNasal inoculationSingle-nucleus RNA sequencingOdor informationNeuronal biologyNeuronsModel diseaseSerotypesConfocal imagingVolatile odorantsRNA sequencingSensory inputAAV1AAVSingle-nucleusTransductionTherapyTropismP-912. A 10-Year Retrospective, Single-Center Analysis of Nocardiosis in Immunocompetent and Immunocompromised Hosts: Clinical and Microbiological Features, Treatment Outcomes, and Determinants of Central Nervous System Involvement
Roman F, Grubman A, Azar M, Farhadian S. P-912. A 10-Year Retrospective, Single-Center Analysis of Nocardiosis in Immunocompetent and Immunocompromised Hosts: Clinical and Microbiological Features, Treatment Outcomes, and Determinants of Central Nervous System Involvement. Open Forum Infectious Diseases 2025, 12: ofae631.1103. PMCID: PMC11778850, DOI: 10.1093/ofid/ofae631.1103.Peer-Reviewed Original ResearchCentral nervous system involvementCentral nervous systemCNS involvementHost immune statusImmune statusImmunocompromised hostsUnivariate analysisAssociated with CNS involvementCentral nervous system nocardiosisRecipients of solid organ transplantsRisk of CNS involvementNet state of immunosuppressionAssociated with central nervous systemIdentification of Nocardia sppMultivariate logistic regression analysisSingle-center analysisStem cell transplantationState of immunosuppressionAssociated with increased riskCNS diseaseIndependent risk factorSolid organ transplantationNervous system involvementIsolates of NocardiaLogistic regression analysisQuantifying VMAT2 target occupancy at effective valbenazine doses and comparing to a novel VMAT2 inhibitor: a translational PET study
Terry-Lorenzo R, Albrecht D, Crouch S, Wong R, Loewen G, Giri N, Skor H, Lin K, Sandiego C, Pajonas M, Rabiner E, Gunn R, Russell D, Haubenberger D. Quantifying VMAT2 target occupancy at effective valbenazine doses and comparing to a novel VMAT2 inhibitor: a translational PET study. Neuropsychopharmacology 2025, 1-9. PMID: 39757283, DOI: 10.1038/s41386-024-02046-3.Peer-Reviewed Original ResearchVesicular monoamine transporter type 2Treatment of tardive dyskinesiaPositron emission tomographyVesicular monoamine transporter type 2 inhibitorsTarget occupancyTreatment of chorea associated with Huntington's diseaseTreatment of TDChorea associated with Huntington's diseaseCentral nervous systemTardive dyskinesiaVMAT2 inhibitorsValbenazineClinical developmentEffect sizeAssociated with efficacyPET studiesEmission tomographyGold standard biomarkerDosing regimensClinical benefitDaily doseHuntington's diseaseActive metabolitePlasma concentrationsAcceptable dose3 Cerebrospinal Fluid
Al Bizri E, Benveniste H. 3 Cerebrospinal Fluid. 2025, 67-76. DOI: 10.1016/b978-0-323-93273-8.00003-1.Peer-Reviewed Original ResearchCurrent Advances in PARP1‐Targeted Theranostics
Tong J, Chen B, Volpi T, Li Y, Ellison P, Cai Z. Current Advances in PARP1‐Targeted Theranostics. Journal Of Labelled Compounds And Radiopharmaceuticals 2025, 68: e4135. PMID: 39995212, DOI: 10.1002/jlcr.4135.Peer-Reviewed Original ResearchConceptsPoly (ADP-ribose) polymerase 1Blood-brain barrierCentral nervous systemBlood-tumor barrierPotential clinical useBRCA mutationsHomologous recombination repair pathwayPARP inhibitorsPharmacodynamic evaluationPatient stratificationPersonalized oncologyPharmacological characteristicsClinical useBrain tumorsBrain barrierDiagnostic imagingPET imagingCancerNervous systemTherapeutic interventionsBrain cancerPathophysiological conditionsTreatment assessmentPARPiNeurological disorders
2024
Bioinformatics analysis of myelin-microbe interactions suggests multiple types of molecular mimicry in the pathogenesis of multiple sclerosis
Bigdeli A, Ghaderi-Zefrehei M, Lesch B, Behmanesh M, Arab S. Bioinformatics analysis of myelin-microbe interactions suggests multiple types of molecular mimicry in the pathogenesis of multiple sclerosis. PLOS ONE 2024, 19: e0308817. PMID: 39775333, PMCID: PMC11684644, DOI: 10.1371/journal.pone.0308817.Peer-Reviewed Original ResearchConceptsBile salt hydrolaseMyelin oligodendrocyte glycoproteinBioinformatics approachProtein structureMyelin basic proteinIn silico bioinformatics approachesAmino acidsIdentical amino acidsAmino acid sequenceCapsid protein structureCentral nervous systemAspergillus speciesAcid sequenceBacterial proteinsMultiple sclerosisMyelin sheath componentsBiological insightsGut flora metabolitesBioinformatics analysisMOG proteinPathogenesis of multiple sclerosisCNS diseaseWzyBacteriaProteinA Systematic Review of Human Paenibacillus Infections and Comparison of Adult and Pediatric Cases.
Smith D, Bastug K, Burgoine K, Broach J, Hammershaimb E, Hehnly C, Morton S, Osman M, Schiff S, Ericson J. A Systematic Review of Human Paenibacillus Infections and Comparison of Adult and Pediatric Cases. The Pediatric Infectious Disease Journal 2024 PMID: 39705610, DOI: 10.1097/inf.0000000000004668.Peer-Reviewed Original ResearchClinical presentation of infectionSurgical management of hydrocephalusPresentation of infectionManagement of hydrocephalusEpidemiology of infectionCentral nervous systemNeonatal infectionBrain abscessSepsis syndromeClinical presentationPediatric casesSurgical managementRare involvementCystic encephalomalaciaAdult patientsInfant casesPatient populationClinical syndromeHydrocephalusCerebral destructionInfectionNervous systemInfantsSystematic reviewBrain destructionAdverse cardiovascular events and cardiac imaging findings in patients on immune checkpoint inhibitors
Kwan J, Shen M, Akhlaghi N, Hu J, Mora R, Cross J, Jiang M, Mankbadi M, Wang P, Zaman S, Lee S, Im Y, Feher A, Liu Y, S. S, Tao W, Wei W, Baldassarre L. Adverse cardiovascular events and cardiac imaging findings in patients on immune checkpoint inhibitors. PLOS ONE 2024, 19: e0314555. PMID: 39621799, PMCID: PMC11611253, DOI: 10.1371/journal.pone.0314555.Peer-Reviewed Original ResearchConceptsAdverse cardiovascular eventsICI therapyCardiac magnetic resonanceIncreased risk of mortalityICI initiationIncreased riskRisk of mortalityCentral nervous systemVentricular dysfunctionCancer patients treated with ICIsCardiovascular eventsLeft ventricular late gadolinium enhancementMortality benefitCardiovascular riskAbnormal left ventricular ejection fractionPatients treated with ICIsLeft ventricular ejection fractionCentral nervous system malignanciesRisk of adverse cardiovascular eventsImmune checkpoint inhibitorsRight ventricular dysfunctionGlobal longitudinal strainVentricular ejection fractionLeft ventricular dysfunctionMultimodality cardiac imagingReasons for screening failure in the evoke and evoke+ trials of semaglutide for early AD
Sano M, Cummings J, Feldman H, Hansson O, van der Flier W, Bardtrum L, Jeppesen R, Johannsen P, Léon T, Hansen C, Scheltens P. Reasons for screening failure in the evoke and evoke+ trials of semaglutide for early AD. Alzheimer's & Dementia 2024, 20: e088735. PMCID: PMC11713595, DOI: 10.1002/alz.088735.Peer-Reviewed Original ResearchMini-Mental State ExaminationAmyloid positivityStrategic infarctsMild AD dementiaRepeatable BatteryPsychiatric disordersCognitive Assessment scoreMild cognitive impairment due to ADInclusion of participantsCognitive impairment due to ADCognitive scoresMini-MentalState ExaminationCDR scoreNeurological disordersScreening failureDisordersEvidence of neurologic disordersAD dementiaScreening failure rateSmall vessel pathologyEvokedScoresCentral nervous systemHierarchical screening procedureIntrathecal lactate dehydrogenase A inhibitors FX11 and oxamate alleviate chronic constriction injury-induced nociceptive sensitization through neuroinflammation and angiogenesis
Cheng H, Chen N, Chen W, Wu Z, Sun Y, Teng W, Su F, Sung C, Wen Z. Intrathecal lactate dehydrogenase A inhibitors FX11 and oxamate alleviate chronic constriction injury-induced nociceptive sensitization through neuroinflammation and angiogenesis. The Journal Of Headache And Pain 2024, 25: 207. PMID: 39587478, PMCID: PMC11590346, DOI: 10.1186/s10194-024-01916-x.Peer-Reviewed Original ResearchConceptsLactate dehydrogenase ANeuropathic painNociceptive sensitizationLDHA inhibitorsTreat neuropathic painType of painUpregulation of glycolysisCCI-induced neuroinflammationCentral nervous systemBackgroundNeuropathic painNeuropathic ratsSpinal roleNociceptive behaviorBlood vessel growthImmunohistochemical analysisControl inflammationPainEffective treatmentConclusionIn conclusionHIF-1aCellular mechanismsNervous systemTherapeutic targetVessel growthWestern blottingQuantification of cinpanemab (BIIB054) binding to α-synuclein in cerebrospinal fluid of phase 1 single ascending dose samples
Liu Y, Yang M, Fraser K, Graham D, Weinreb P, Weihofen A, Hirst W, Cedarbaum J, Pepinsky B. Quantification of cinpanemab (BIIB054) binding to α-synuclein in cerebrospinal fluid of phase 1 single ascending dose samples. Journal Of Pharmacology And Experimental Therapeutics 2024, 392: 100003. PMID: 39892989, DOI: 10.1124/jpet.124.002199.Peer-Reviewed Original ResearchCentral nervous systemCerebrospinal fluidMeso Scale DiscoveryCentral nervous system compartmentDrug concentrationsA-synCerebrospinal fluid samplesParkinson's diseasePassive immunotherapy approachesSite of actionImmunotherapy approachesLow drug concentrationsImmunotherapy trialsHealthy volunteersSystemic compartmentDrug-target interactionsCSF samplesObserved doseNervous systemTherapeutic targetA-syn levelsClinical samplesTime-dependent bindingComplex formationCross-linking methodR-Spondin 1 Suppresses Inflammatory Cytokine Production in Human Cortical Astrocytes
Logan R, Bhatta S, Sutova H, Hafler B, Miller S. R-Spondin 1 Suppresses Inflammatory Cytokine Production in Human Cortical Astrocytes. Neuroglia 2024, 5: 445-451. DOI: 10.3390/neuroglia5040028.Peer-Reviewed Original ResearchCentral nervous systemCytokine releaseSignaling pathwayCentral nervous system inflammationR-spondinStem cell-derived astrocytesInfluence central nervous systemHuman induced pluripotent stem cell-derived astrocytesCytokine array kitSuppression of inflammatory cytokinesModulate cytokine releaseWnt signalingInfluence inflammatory responsesModulate immune responsesInflammatory cytokine productionCytokine-cytokine receptor interactionInflammatory cytokine releaseContext of inflammationLGR6 receptorsPathway enrichment analysisCXCL12 levelsHuman cortical astrocytesIL-23TNF signaling pathwayIFN-gThe Incidence of CNS Relapse in Philadelphia Chromosome Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) and Its Impact on Clinical Outcome: Results from Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Shah K, El Kettani M, Narra R, Mims A, Coltoff A, Medawar G, Hisrich B, Shallis R, Hunter C, Kota V, Othman T, Jonas B, Desai S, Sacchi de Camargo Correia G, Patel A, Duvall A, Palmisiano N, Curran E, Omer Z, Advani A, Atallah E, Litzow M, Badar T. The Incidence of CNS Relapse in Philadelphia Chromosome Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) and Its Impact on Clinical Outcome: Results from Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Blood 2024, 144: 4184-4184. DOI: 10.1182/blood-2024-205812.Peer-Reviewed Original ResearchCNS relapseCentral nervous system involvementPh+ acute lymphoblastic leukemiaRelapse free survivalTyrosine kinase inhibitorsAcute lymphoblastic leukemiaIncidence of CNS relapseMedian relapse free survivalCentral nervous systemCNS diseaseOverall survivalAllo-HCTIntrathecal chemotherapyWhite blood cellsIntensive chemotherapyPost-relapseT315I mutationBaseline characteristicsMedian time to CNS relapseAllogeneic stem cell transplantationAssociated with significant toxicityIncidence of CNS diseaseMedian white blood cellAdequate CNS prophylaxisCentral nervous system penetrationNeuroprotective effect of neuron‐specific deletion of the C16 ceramide synthetic enzymes in an animal model of multiple sclerosis
Amatruda M, Marechal D, Gacias M, Wentling M, Turpin‐Nolan S, Morstein J, Moniruzzaman M, Brüning J, Haughey N, Trauner D, Casaccia P. Neuroprotective effect of neuron‐specific deletion of the C16 ceramide synthetic enzymes in an animal model of multiple sclerosis. Glia 2024, 73: 271-290. PMID: 39489703, PMCID: PMC11662985, DOI: 10.1002/glia.24631.Peer-Reviewed Original ResearchExperimental autoimmune encephalomyelitisNeuron-specific deletionMultiple sclerosisDemyelinating disordersCourse of EAESalvage pathwayAnimal model of multiple sclerosisModel of multiple sclerosisDisease severityRecycling of sphingosineWild type miceCeramide synthase 6Central nervous systemPalmitic acidAutoimmune encephalomyelitisExposure to oxidative stressType miceInflammatory demyelinationMyelin damageSaturated fatAnimal modelsMitochondrial dysfunctionCerS5Nervous systemNeuroprotective effects
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