Use of genome editing tools in human stem cell-based disease modeling and precision medicine.
Precision medicine emerges as a new approach that takes into account individual variability. The successful conduct of precision medicine requires the use of precise disease models. Human pluripotent stem cells (hPSCs), as well as adult stem cells, can be differentiated into a variety of human somatic cell types that can be used for research and drug screening. The development of genome editing technology over the past few years, especially the CRISPR/Cas system, has made it feasible to precisely and efficiently edit the genetic background. Therefore, disease modeling by using a combination of human stem cells and genome editing technology has offered a new platform to generate " personalized " disease models, which allow the study of the contribution of individual genetic variabilities to disease progression and the development of precise treatments. In this review, recent advances in the use of genome editing in human stem cells and the generation of stem cell models for rare diseases and cancers are discussed.
Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
Yu-da
YD
Wei
Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
Shuang
S
Li
Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
Gai-gai
GG
Liu
Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
Yong-xian
YX
Zhang
Institute for Nutritional Sciences, Shanghai Institutes for Biological Sciences, Shanghai 200031, China.
Qiu-rong
QR
Ding
D064113:CRISPR-Cas Systems; D004194:Disease / Q000235:genetics*; D005818:Genetic Engineering / Q000379:methods*; D015894:Genome, Human / Q000235:genetics*; D006801:Humans; D008957:Models, Genetic; D009369:Neoplasms / Q000235:genetics / Q000473:pathology; D057285:Precision Medicine / Q000379:methods* / Q000639:trends; D035583:Rare Diseases / Q000235:genetics / Q000473:pathology; D013234:Stem Cells / Q000378:metabolism*