Phenotypic Screening Using Patient-Derived Induced Pluripotent Stem Cells Identified Pyr3 as a Candidate Compound for the Treatment of Infantile Hypertrophic Cardiomyopathy
Sakai T, Naito AT, Kuramoto Y, Ito M, Okada K, Higo T, Nakagawa A, Shibamoto M, Yamaguchi T, Sumida T, Nomura S, Umezawa A, Miyagawa S, Sawa Y, Morita H, Lee JK, Shiojima I, Sakata Y, Komuro I. Phenotypic Screening Using Patient-Derived Induced Pluripotent Stem Cells Identified Pyr3 as a Candidate Compound for the Treatment of Infantile Hypertrophic Cardiomyopathy. International Heart Journal 2018, 59: 17-730. PMID: 30101858, DOI: 10.1536/ihj.17-730.Peer-Reviewed Original ResearchMeSH KeywordsAdultCalciumCardiomyopathy, HypertrophicChild, PreschoolHumansInduced Pluripotent Stem CellsMaleMass ScreeningMutationMyocardiumMyocytes, CardiacNoonan SyndromePhenotypePrevalenceTransient Receptor Potential ChannelsConceptsDiastolic intracellular calcium concentrationInfantile hypertrophic cardiomyopathyHypertrophic cardiomyopathyIntracellular calcium concentrationHCM patientsIPSC-CMsInduced pluripotent stem cellsNoonan syndromeCalcium concentrationIdiopathic hypertrophic cardiomyopathyPatient-derived induced pluripotent stem cellsStem cellsPluripotent stem cellsHealthy subjectsPatientsChannel inhibitorsPhenotypic screeningPyr3Genetic disordersDisease-related phenotypesCardiomyopathySyndromeCandidate compoundsPresent studyTreatment