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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Published in | CRISPR journal Vol. 6; no. 3; p. 196 |
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Main Authors | , , , , , , |
Format | Journal Article |
Language | English |
Published |
United States
01.06.2023
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Abstract | 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. |
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AbstractList | 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. |
Author | Wong, Poh Kuan Syafruddin, Saiful Effendi Chua, Eng Wee Azmi, Norazrina Law, Jia Xian Mohamad Zamberi, Nurul Nadia Cheah, Fook Choe |
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SubjectTerms | Cell Line CRISPR-Cas Systems - genetics Embryonic Stem Cells - metabolism Gene Editing Humans Induced Pluripotent Stem Cells - metabolism |
Title | The Promises and Pitfalls of CRISPR-Mediated Base Editing in Stem Cells |
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