The Promises and Pitfalls of CRISPR-Mediated Base Editing in Stem Cells

Stem cells such as induced pluripotent stem cells, embryonic stem cells, and hematopoietic stem and progenitor cells are growing in importance in disease modeling and regenerative medicine. The applications of CRISPR-based gene editing to create a mélange of disease and nondisease stem cell lines ha...

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Bibliographic Details
Published inCRISPR journal Vol. 6; no. 3; p. 196
Main Authors Wong, Poh Kuan, Mohamad Zamberi, Nurul Nadia, Syafruddin, Saiful Effendi, Cheah, Fook Choe, Azmi, Norazrina, Law, Jia Xian, Chua, Eng Wee
Format Journal Article
LanguageEnglish
Published United States 01.06.2023
Subjects
Cell Line
CRISPR-Cas Systems - genetics
Embryonic Stem Cells - metabolism
Gene Editing
Humans
Induced Pluripotent Stem Cells - metabolism
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Summary:Stem cells such as induced pluripotent stem cells, embryonic stem cells, and hematopoietic stem and progenitor cells are growing in importance in disease modeling and regenerative medicine. The applications of CRISPR-based gene editing to create a mélange of disease and nondisease stem cell lines have further enhanced the utility of this innately versatile group of cells in the studies of human genetic disorders. Precise base edits can be achieved using a variety of CRISPR-centric approaches, particularly homology-directed repair and the recently developed base editors and prime editors. Despite its much-touted potential, editing single DNA bases is technically challenging. In this review, we discuss the strategies for achieving exact base edits in the creation of various stem cell-based models for use in elucidating disease mechanisms and assessing drug efficacy, and the unique characteristics of stem cells that warrant special considerations.
ISSN:2573-1602
DOI:10.1089/crispr.2023.0013