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

被引：3

作者：

Wong, Poh Kuan ^{[1
]}

Zamberi, Nurul Nadia Mohamad ^{[2
]}

Syafruddin, Saiful Effendi ^{[2
]}

Cheah, Fook Choe ^{[3
]}

Azmi, Norazrina ^{[1
]}

Law, Jia Xian ^{[4
]}

Chua, Eng Wee ^{[1
]}

机构：

[1] Univ Kebangsaan Malaysia, Fac Pharm, Ctr Drug & Herbal Dev, Jalan Raja Muda Abdul Aziz, Kuala Lumpur 50300, Malaysia

[2] Univ Kebangsaan Malaysia, UKM Med Mol Biol Inst, Kuala Lumpur, Malaysia

[3] Univ Kebangsaan Malaysia, Med Ctr, Dept Paediat, Kuala Lumpur, Malaysia

[4] Univ Kebangsaan Malaysia, Fac Med, Ctr Tissue Engn & Regenerat Med, Kuala Lumpur, Malaysia

来源：

CRISPR JOURNAL | 2023年 / 6卷 / 03期

关键词：

HIGHLY EFFICIENT; MESSENGER-RNA; IN-VIVO; GENOMIC DNA; DELIVERY; CAS9; GENE; MUTATIONS; REPAIR; IMMUNOGENICITY;

D O I：

10.1089/crispr.2023.0013

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

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 melange 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.

引用

页码：196 / 215

页数：20

共 151 条

[1] Reverting TP53 Mutation in Breast Cancer Cells: Prime Editing Workflow and Technical Considerations [J].