Human induced pluripotent stem cells and neurodegenerative disease
Jung YW, Hysolli E, Kim KY, Tanaka Y, Park IH. Human induced pluripotent stem cells and neurodegenerative disease. Current Opinion In Neurology 2012, 25: 125-130. PMID: 22357218, PMCID: PMC3786112, DOI: 10.1097/wco.0b013e3283518226.Peer-Reviewed Original ResearchConceptsNeurodegenerative diseasesReplacement therapyCell replacement therapyParkinson's diseasePositive therapeutic effectDisease modelsParkinson's disease modelAmyotrophic lateral sclerosisNeuron-specific transcription factorSpinal muscular atrophyStem cellsDrug screeningDopaminergic neuronsIntermediate cell typeTherapeutic effectEffective treatmentLateral sclerosisDisease-specific iPSCsMuscular atrophyAlzheimer's diseaseNeurodegenerative disordersCellular therapyDiseaseTherapyHuman induced pluripotent stem cellsMutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability
Bilican B, Serio A, Barmada SJ, Nishimura AL, Sullivan GJ, Carrasco M, Phatnani HP, Puddifoot CA, Story D, Fletcher J, Park IH, Friedman BA, Daley GQ, Wyllie DJ, Hardingham GE, Wilmut I, Finkbeiner S, Maniatis T, Shaw CE, Chandran S. Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability. Proceedings Of The National Academy Of Sciences Of The United States Of America 2012, 109: 5803-5808. PMID: 22451909, PMCID: PMC3326463, DOI: 10.1073/pnas.1202922109.Peer-Reviewed Original ResearchConceptsAmyotrophic lateral sclerosisTDP-43 proteinopathyTDP-43Human neuronsTransactive response DNA binding proteinFrontotemporal lobar degenerationFamilial amyotrophic lateral sclerosisFunctional motor neuronsCell-specific vulnerabilityTDP-43 proteinDNA binding proteinPI3K pathwayCell-autonomous phenotypeMotor neuronsLateral sclerosisStem cell linesPluripotent stem cellsGeneration of iPSCsIdentification of mutationsPluripotent stem cell lineMutant neuronsNeuronsK pathwayElevated levelsDisease mechanisms