May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells

Cells

Christopher Potts¹  David. A. Brafman¹  Sreedevi Raman¹  Nicholas Brookhouser¹ 

[1] School of Biological and Health Systems Engineering, Arizona State University, 501 E. Tyler Mall, ECG 334A, Tempe, AZ 85287, USA;

关键词: human induced pluripotent stem cells (hiPSCs);    genome editing;    homology-directed repair;    ZFN;    TALEN;    CRISPR/Cas9;   

DOI  :  10.3390/cells6010005

来源: DOAJ

【 摘 要 】

In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods.

【 授权许可】

Unknown