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Gene Editing and Cell Therapy Based on Induced Pluripotent Stem Cells
Yang Wei1, Li Shishi1, Zhang Xuan1, Pan Huaye1, Yan Qingfeng1,2*
1College of Life Sciences, Zhejiang University, Hangzhou 310058, China; 2Institute of Genetics, Zhejiang University, Hangzhou 310058, China)
Abstract: Induced pluripotent stem cells (iPSCs) are embryonic stem cell-like cells with self-renewal and differentiation capacity， which are reprogrammed from adult cells. Gene editing for the specific sites of genome can achieve the targeted genetic modification conveniently and efficiently. With improvement of efficacy and safety in cell reprogramming， gene editing and genetic correction for the pathogenic mutation of iPSCs derived from patients are attracting increasing attention on translational medicine， which open a remarkable avenue for cell therapy. In this review， we summarized the principles of gene editing， and then introduced the advance of gene editing and cell therapy in iPSCs. Occurring challenges and perspectives were also discussed.
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