Genome editing of disease-specific induced pluripotent stem cells

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Reprogramming has enabled the generation of patient-specific induced pluripotent stem (iPS) cells which can be used in disease modeling. However considerable clonal variability exists among different disease-specific iPSCs. One  way to overcome the variability among disease-specific iPSC lines, especially with respect to monogenic diseases, is to generate genetically defined sibling cell lines. Genome editing usingZinc finger nucleases (ZFNs)transcription activator-like effector nucleases (TALENs) and Crispr/Cas Systems have enabled correction of disease causing mutations. We are working to apply these technologies to derive gene corrected iPSCs from a wide variety of monogenic diseases frequetly observed in Turkey.

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