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Disease-specific induced pluripotent stem cells: a platform for human disease modeling and drug discovery.

DC Field Value Language
dc.contributor.author김한수-
dc.contributor.author장지호-
dc.contributor.author허용준-
dc.contributor.author강훈철-
dc.contributor.author김대성-
dc.contributor.author유정은-
dc.contributor.author김동욱-
dc.contributor.author이동진-
dc.contributor.author김지영-
dc.contributor.author이정아-
dc.date.accessioned2014-12-19T16:49:32Z-
dc.date.available2014-12-19T16:49:32Z-
dc.date.issued2012-
dc.identifier.issn1226-3613-
dc.identifier.urihttps://ir.ymlib.yonsei.ac.kr/handle/22282913/90265-
dc.description.abstractThe generation of disease-specific induced pluripotent stem cell (iPSC) lines from patients with incurable diseases is a promising approach for studying disease mechanisms and drug screening. Such innovation enables to obtain autologous cell sources in regenerative medicine. Herein, we report the generation and characterization of iPSCs from fibroblasts of patients with sporadic or familial diseases, including Parkinson's disease (PD), Alzheimer's disease (AD), juvenile-onset, type I diabetes mellitus (JDM), and Duchenne type muscular dystrophy (DMD), as well as from normal human fibroblasts (WT). As an example to modeling disease using disease-specific iPSCs, we also discuss the previously established childhood cerebral adrenoleukodystrophy (CCALD)- and adrenomyeloneuropathy (AMN)-iPSCs by our group. Through DNA fingerprinting analysis, the origins of generated disease-specific iPSC lines were identified. Each iPSC line exhibited an intense alkaline phosphatase activity, expression of pluripotent markers, and the potential to differentiate into all three embryonic germ layers: the ectoderm, endoderm, and mesoderm. Expression of endogenous pluripotent markers and downregulation of retrovirus-delivered transgenes [OCT4 (POU5F1), SOX2, KLF4, and c-MYC] were observed in the generated iPSCs. Collectively, our results demonstrated that disease-specific iPSC lines characteristically resembled hESC lines. Furthermore, we were able to differentiate PD-iPSCs, one of the disease-specific-iPSC lines we generated, into dopaminergic (DA) neurons, the cell type mostly affected by PD. These PD-specific DA neurons along with other examples of cell models derived from disease-specific iPSCs would provide a powerful platform for examining the pathophysiology of relevant diseases at the cellular and molecular levels and for developing new drugs and therapeutic regimens.-
dc.description.statementOfResponsibilityopen-
dc.formatapplication/pdf-
dc.relation.isPartOfEXPERIMENTAL AND MOLECULAR MEDICINE-
dc.rightsCC BY-NC-ND 2.0 KR-
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/2.0/kr/-
dc.subject.MESHAlzheimer Disease/genetics-
dc.subject.MESHAlzheimer Disease/pathology*-
dc.subject.MESHCell Differentiation-
dc.subject.MESHCells, Cultured-
dc.subject.MESHDiabetes Mellitus, Type 1/genetics-
dc.subject.MESHDiabetes Mellitus, Type 1/pathology*-
dc.subject.MESHDrug Discovery/methods*-
dc.subject.MESHFibroblasts/cytology-
dc.subject.MESHFibroblasts/metabolism-
dc.subject.MESHFibroblasts/pathology-
dc.subject.MESHGene Expression-
dc.subject.MESHHumans-
dc.subject.MESHInduced Pluripotent Stem Cells/cytology-
dc.subject.MESHInduced Pluripotent Stem Cells/metabolism-
dc.subject.MESHInduced Pluripotent Stem Cells/pathology*-
dc.subject.MESHMuscular Dystrophy, Duchenne/genetics-
dc.subject.MESHMuscular Dystrophy, Duchenne/pathology*-
dc.subject.MESHParkinson Disease/genetics-
dc.subject.MESHParkinson Disease/pathology*-
dc.titleDisease-specific induced pluripotent stem cells: a platform for human disease modeling and drug discovery.-
dc.typeArticle-
dc.contributor.collegeCollege of Medicine (의과대학)-
dc.contributor.departmentDept. of Physiology (생리학)-
dc.contributor.googleauthorJiho Jang-
dc.contributor.googleauthorJeong-Eun Yoo-
dc.contributor.googleauthorJeong-Ah Lee-
dc.contributor.googleauthorDongjin R. Lee-
dc.contributor.googleauthorJi Young Kim-
dc.contributor.googleauthorYong Jun Huh-
dc.contributor.googleauthorDae-Sung Kim-
dc.contributor.googleauthorChul-Yong Park-
dc.contributor.googleauthorDong-Youn Hwang-
dc.contributor.googleauthorHan-Soo Kim-
dc.contributor.googleauthorHoon-Chul Kang-
dc.contributor.googleauthorDong-Wook Kim-
dc.identifier.doi22179105-
dc.admin.authorfalse-
dc.admin.mappingfalse-
dc.contributor.localIdA01100-
dc.contributor.localIdA03480-
dc.contributor.localIdA04360-
dc.contributor.localIdA00102-
dc.contributor.localIdA00367-
dc.contributor.localIdA02734-
dc.contributor.localIdA03113-
dc.contributor.localIdA00981-
dc.contributor.localIdA02505-
dc.contributor.localIdA00406-
dc.relation.journalcodeJ00860-
dc.identifier.eissn2092-6413-
dc.identifier.pmid22179105-
dc.subject.keywordAlzheimer Disease/genetics-
dc.subject.keywordAlzheimer Disease/pathology*-
dc.subject.keywordCell Differentiation-
dc.subject.keywordCells, Cultured-
dc.subject.keywordDiabetes Mellitus, Type 1/genetics-
dc.subject.keywordDiabetes Mellitus, Type 1/pathology*-
dc.subject.keywordDrug Discovery/methods*-
dc.subject.keywordFibroblasts/cytology-
dc.subject.keywordFibroblasts/metabolism-
dc.subject.keywordFibroblasts/pathology-
dc.subject.keywordGene Expression-
dc.subject.keywordHumans-
dc.subject.keywordInduced Pluripotent Stem Cells/cytology-
dc.subject.keywordInduced Pluripotent Stem Cells/metabolism-
dc.subject.keywordInduced Pluripotent Stem Cells/pathology*-
dc.subject.keywordMuscular Dystrophy, Duchenne/genetics-
dc.subject.keywordMuscular Dystrophy, Duchenne/pathology*-
dc.subject.keywordParkinson Disease/genetics-
dc.subject.keywordParkinson Disease/pathology*-
dc.contributor.alternativeNameKim, Han Soo-
dc.contributor.alternativeNameJang, Ji Ho-
dc.contributor.alternativeNameHuh, Yong Jun-
dc.contributor.alternativeNameKang, Hoon Chul-
dc.contributor.alternativeNameKim, Dae Sung-
dc.contributor.alternativeNameYoo, Jeong Eun-
dc.contributor.alternativeNameKim, Dong Wook-
dc.contributor.alternativeNameLee, Dongjin R.-
dc.contributor.alternativeNameKim, Ji Young-
dc.contributor.alternativeNameLee, Jeong Ah-
dc.contributor.affiliatedAuthorKim, Han Soo-
dc.contributor.affiliatedAuthorJang, Ji Ho-
dc.contributor.affiliatedAuthorHuh, Yong Jun-
dc.contributor.affiliatedAuthorKang, Hoon Chul-
dc.contributor.affiliatedAuthorKim, Dae Sung-
dc.contributor.affiliatedAuthorLee, Dongjin R.-
dc.contributor.affiliatedAuthorLee, Jeong Ah-
dc.contributor.affiliatedAuthorKim, Ji Young-
dc.contributor.affiliatedAuthorYoo, Jeong Eun-
dc.contributor.affiliatedAuthorKim, Dong Wook-
dc.citation.volume44-
dc.citation.number3-
dc.citation.startPage202-
dc.citation.endPage213-
dc.identifier.bibliographicCitationEXPERIMENTAL AND MOLECULAR MEDICINE, Vol.44(3) : 202-213, 2012-
dc.identifier.rimsid34104-
dc.type.rimsART-
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Laboratory Medicine (진단검사의학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Physiology (생리학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Yonsei Biomedical Research Center (연세의생명연구원) > 1. Journal Papers

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