2020
Implantation of the clinical‐grade human neural stem cell line, CTX0E03, rescues the behavioral and pathological deficits in the quinolinic acid‐lesioned rodent model of Huntington's disease
Yoon Y, Kim HS, Jeon I, Noh J, Park HJ, Lee S, Park I, Stevanato L, Hicks C, Corteling R, Barker RA, Sinden JD, Song J. Implantation of the clinical‐grade human neural stem cell line, CTX0E03, rescues the behavioral and pathological deficits in the quinolinic acid‐lesioned rodent model of Huntington's disease. Stem Cells 2020, 38: 936-947. PMID: 32374064, PMCID: PMC7496241, DOI: 10.1002/stem.3191.Peer-Reviewed Original ResearchConceptsMedium spiny neuronsNeural stem cell lineHuntington's diseaseQuinolinic acid (QA) lesion rat modelChronic ischemic stroke patientsStriatal medium spiny neuronsCell linesImmortalized neural stem cell linesIschemic stroke patientsDisease-modifying therapiesSignals of efficacyGlial scar formationHost brain tissueHuman neural stem cell lineSignificant behavioral improvementAutosomal dominant neurodegenerative diseaseCTX0E03 cellsEndogenous neurogenesisBDNF expressionGABAergic neuronsHD patientsStroke patientsFluoro-GoldRetrograde labelSpiny neurons
2014
In Vivo Roles of a Patient-Derived Induced Pluripotent Stem Cell Line (HD72-iPSC) in the YAC128 Model of Huntington’s Disease
Jeon I, Choi C, Lee N, Im W, Kim M, Oh SH, Park IH, Kim HS, Song J. In Vivo Roles of a Patient-Derived Induced Pluripotent Stem Cell Line (HD72-iPSC) in the YAC128 Model of Huntington’s Disease. International Journal Of Stem Cells 2014, 7: 43-47. PMID: 24921027, PMCID: PMC4049731, DOI: 10.15283/ijsc.2014.7.1.43.Peer-Reviewed Original ResearchHuntington's diseaseHD pathologyNeural precursorsAvailable therapeutic optionsHD mouse modelsYAC128 transgenic miceSignificant behavioral improvementCAG repeatsVivo roleYAC128 modelGrafted miceTherapeutic optionsGrafted cellsPatient-derived iPSCsMouse modelTransgenic miceBehavioral improvementDiseaseNovel therapeuticsMiceCell linesPluripotent stem cell linePathologyStem cellsNeurodegenerative genetic disease
2012
Neuronal Properties, In Vivo Effects, and Pathology of a Huntington's Disease Patient‐Derived Induced Pluripotent Stem Cells
Jeon I, Lee N, Li J, Park I, Park KS, Moon J, Shim SH, Choi C, Chang D, Kwon J, Oh S, Shin DA, Kim HS, Tae J, Lee DR, Kim M, Kang K, Daley GQ, Brundin P, Song J. Neuronal Properties, In Vivo Effects, and Pathology of a Huntington's Disease Patient‐Derived Induced Pluripotent Stem Cells. Stem Cells 2012, 30: 2054-2062. PMID: 22628015, DOI: 10.1002/stem.1135.Peer-Reviewed Original ResearchConceptsHD-iPSCHD pathologyHuntington's diseaseDisease patientsNeuronal propertiesUnilateral excitotoxic striatal lesionExcitotoxic striatal lesionsSignificant behavioral recoveryStem cellsGABAergic striatal neuronsHuntington's disease patientsCAG repeatsNeuronal cell typesPluripotent stem cellsBehavioral recoveryGABAergic neuronsStriatal lesionsStriatal neuronsRat modelNeonatal brainNovel cell therapiesVivo effectsHD phenotypeCell therapyNovel therapeutics
2008
Disease-Specific Induced Pluripotent Stem Cells
Park IH, Arora N, Huo H, Maherali N, Ahfeldt T, Shimamura A, Lensch MW, Cowan C, Hochedlinger K, Daley GQ. Disease-Specific Induced Pluripotent Stem Cells. Cell 2008, 134: 877-886. PMID: 18691744, PMCID: PMC2633781, DOI: 10.1016/j.cell.2008.07.041.Peer-Reviewed Original ResearchConceptsParkinson's diseaseDown syndromeBecker muscular dystrophyType 1 diabetes mellitusHuntington's diseaseStem cellsPluripotent stem cellsDiabetes mellitusDisease-SpecificLesch-Nyhan syndromeDisease-specific stem cellsDiseased patientsCarrier stateTumor cell linesDiseaseSyndromeMuscular dystrophyAdenosine deaminasePatientsDrug developmentInduced pluripotent stem cellsType IIICell linesDiamond syndromeDisease investigation