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- W3136196304 abstract "The study of human genetic disorders using animal models has been a mainstay of biomedical research for decades. Recent advances in stem cell biology have enabled an alternative approach to model human disorders—the use of human-derived cells. When paired with modern genome-editing tools, human-derived cells offer the ability to study human diseases with unprecedented granularity, deconstructing molecular pathophysiology one gene or variant at a time. Although many different types of human-derived cells are amenable to genome editing and are available for use by investigators, including primary human cells and organoids as well as transformed or immortalized cultured cell lines, the focus of this chapter is on the use of human pluripotent stem cells (hPSCs), particularly induced pluripotent stem cells. That notwithstanding, the lessons from studies of hPSCs illustrated herein are broadly relevant to all human cell types and, indeed, cells of any species being employed for experimental research." @default.
- W3136196304 created "2021-03-29" @default.
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- W3136196304 date "2021-01-01" @default.
- W3136196304 modified "2023-09-23" @default.
- W3136196304 title "Genome editing for cellular disease modeling" @default.
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- W3136196304 doi "https://doi.org/10.1016/b978-0-12-823484-6.00002-5" @default.
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