2023
In Utero Electroporated Neurons for Medium-Throughput Screening of Compounds Regulating Neuron Morphology
Sokolov A, Aurich M, Bordey A. In Utero Electroporated Neurons for Medium-Throughput Screening of Compounds Regulating Neuron Morphology. ENeuro 2023, 10: eneuro.0160-23.2023. PMID: 37620147, PMCID: PMC10464655, DOI: 10.1523/eneuro.0160-23.2023.Peer-Reviewed Original ResearchConceptsSomatosensory cortexCortical pyramidal neuronsTreatment of epilepsyNeurite overgrowthNeurologic disabilityPyramidal neuronsSoma sizeNovel agentsCircuit alterationsSide effectsMorphologic assessmentMouse neuronsRelated disordersDiseased neuronsMTOR activatorDrug efficacyCandidate therapeuticsNeuronsNeuron morphologyMTOR activityMedium-throughput screeningNeurodevelopmental disordersNeurite lengthMorphologic measurementsDisorders
2020
Pharmacological reversal of synaptic and network pathology in human MECP2‐KO neurons and cortical organoids
Trujillo CA, Adams JW, Negraes PD, Carromeu C, Tejwani L, Acab A, Tsuda B, Thomas CA, Sodhi N, Fichter KM, Romero S, Zanella F, Sejnowski TJ, Ulrich H, Muotri AR. Pharmacological reversal of synaptic and network pathology in human MECP2‐KO neurons and cortical organoids. EMBO Molecular Medicine 2020, 13: emmm202012523. PMID: 33501759, PMCID: PMC7799367, DOI: 10.15252/emmm.202012523.Peer-Reviewed Original ResearchConceptsRett syndromeCortical organoidsPredominant etiologyNeurodevelopmental impairmentPharmacological reversalPHA-543613Neuropathologic phenotypeSynaptic dysregulationClinical studiesHuman pluripotent stem cell technologySymptomatic severityHuman neuronsMeCP2 deficiencyCandidate therapeuticsBrain mosaicismNetwork pathologyPharmacological compoundsPluripotent stem cell (iPSC) technologyNeurodevelopmental disordersMECP2 mutationsNeuropathologyMECP2 geneNeuronsCellular mosaicismStem cell technology645 Preclinical development of a novel colon-targeted therapeutic for the treatment of immune checkpoint inhibitor (ICI)-colitis
Dizman N, Greenberg M, Newman A, Skupsky J, Hsu J, Zengin Z, Salgia N, Meza L, Chawla N, Salgia S, Malhotra J, Pal S. 645 Preclinical development of a novel colon-targeted therapeutic for the treatment of immune checkpoint inhibitor (ICI)-colitis. Journal For ImmunoTherapy Of Cancer 2020, 8: a682-a682. DOI: 10.1136/jitc-2020-sitc2020.0645.Peer-Reviewed Original ResearchICI therapySide effectsSystemic inflammationT cellsImmune checkpoint inhibitor therapyFrequent dose-limiting toxicityCandidate therapeuticsCenter Institutional Review BoardFavorable drug profileSigns of colitisCheckpoint inhibitor therapyImmune checkpoint inhibitorsDose-limiting toxicityMinimal systemic absorptionRegulatory T cellsAdoptive transfer modelAnti-inflammatory effectsSite of ulcerationT cell proliferationLife-threatening cancerRodent pharmacokinetic studyCancer-killing abilityInstitutional review boardActive colitisICI colitis
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