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Disease Specific Induced Pluripotent Stem Cells by Using Transcription Factors

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.