전사인자를 이용한 질병특이 유도전능줄기세포
Disease Specific Induced Pluripotent Stem Cells by Using Transcription Factors
1
조회
0
다운로드
0
인용
0
참고문헌
키워드
초록
The recent years have seen great advances in reversal of programming of differentiated somatic cells towards pluripotency by methods not involving nuclear transfer. Although differentiation of cells involves complex genetic and epigenetic changes, it is now possible to generate cells with many properties of pluripotent embryonic stem cells by transduction of differentiated cells with only four transcription factors: Oct3/4, Sox2, Klf4 and c-Myc. In advance, the protocol for improving the efficiency of generating induced pluripotent stem (iPS) cells and establishing clinically applicable cells has been developed. Recently, the generation of iPS cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance including X-linked adrenoleukodystrophy, amyotrophic lateral sclerosis, Duchenne and Becker muscular dystrophy, Parkinson's disease, Huntington's disease, Gaucher disease type III, Down syndrome is also possible. Such disease-specific iPS cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation, drug development and cell therapy.
Abstract
The recent years have seen great advances in reversal of programming of differentiated somatic cells towards pluripotency by methods not involving nuclear transfer. Although differentiation of cells involves complex genetic and epigenetic changes, it is now possible to generate cells with many properties of pluripotent embryonic stem cells by transduction of differentiated cells with only four transcription factors: Oct3/4, Sox2, Klf4 and c-Myc. In advance, the protocol for improving the efficiency of generating induced pluripotent stem (iPS) cells and establishing clinically applicable cells has been developed. Recently, the generation of iPS cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance including X-linked adrenoleukodystrophy, amyotrophic lateral sclerosis, Duchenne and Becker muscular dystrophy, Parkinson's disease, Huntington's disease, Gaucher disease type III, Down syndrome is also possible. Such disease-specific iPS cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation, drug development and cell therapy.
참고문헌 (18)
- Sell S, "Stem cells: What are they? Where do they come from? Why are they here? When do they go wrong? Where are they going? in Stem cells handbook. 1st ed", pp.1~18 (2004), Humana Press Co
- Kirschstein R, "The stem cell in Stem cells: scientific progress and future research directions. 1st ed", pp.1~4 (2001), National Institutes of Health
- Lewitzky M, "Reprogramming somatic cells towards pluripotency by defined factors", Curr Opin Biotechnol, 18, pp.467~473 (2007)
- Evans MJ, "Mitochondrial DNA genotypes in nuclear transfer-derived cloned sheep", Nat Genet, 23, pp.90~93 (1999)
- Thomson JA, "Embryonic stem cell lines derived from human blastocysts", Science, 282, pp.1145~1147 (1998)
- Takahashi K, "Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined fa", Cell, 126, pp.663~676 (2006)
- Chang MY, "Direct reprogramming of rat neural precursor cells and fibroblasts into pluripotent stem cells", PLoS One, 5, pp.e9838~e9847 (2010)
- Takahashi K, "Induction of pluripotent stem cells from adult human fibroblasts by defined factors", Cell, 131, pp.861~872 (2007)
- Park IH, "Diseasespecific induced pluripotent stem cells", Cell, 134, pp.877~886 (2008)
- Yu J, "Induced pluripotent stem cell lines derived from human somatic cells", Science, 318, pp.1917~1920 (2007)
- Kim JB, "Direct reprogramming of human neural stem cells by OCT4", Nature, 461, pp.649~643 (2009)
- Mali P, "Improved efficiency and pace of generating induced pluripotent stem cells from human adult and fetal fibroblasts", Stem Cells, 26, pp.1998~2005 (2008)
- Schugar RC, "Small molecules in stem cell self-renewal and differentiation", Gene Ther, 15, pp.126~135 (2008)
- Kim D, "Generation of human induced pluripotent stem cells by dire delivery of reprogramming proteins", Cell Stem Cell, 4, pp.472~476 (2009)
- Soldner F, "Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors", Cell, 136, pp.964~977 (2009)
- Moser HW, "Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy", Brain, 120, pp.1485~1508 (1997)
- Kemp S, "X-linked adrenoleukodystrophy: very long-chain fatty acid metabolism, ABC half-transporters and the complicated route to treatment", Mol Genet Metab, 90, pp.268~276 (2007)
- Semmler A, "Therapy of X-linked adrenoleukodystrophy", Expert Rev Neurother, 8, pp.1367~1379 (2008)