Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells

Malihe Lotfi; Dorsa Morshedi Rad; Samaneh Sharif Mashhadi; Atefeh Ashouri; Majid Mojarrad; Sina Mozaffari-Jovin; Shima Farrokhi; Maryam Hashemi; Marzieh Lotfi; Majid Ebrahimi Warkiani; Mohammad Reza Abbaszadegan

doi:10.1007/s12015-023-10585-3

Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells

Stem Cell Rev Rep. 2023 Nov;19(8):2576-2596. doi: 10.1007/s12015-023-10585-3. Epub 2023 Sep 18.

Authors

Malihe Lotfi^#^{1

2

3}, Dorsa Morshedi Rad^#⁴, Samaneh Sharif Mashhadi^{2

3}, Atefeh Ashouri^{2

3}, Majid Mojarrad^{2

3}, Sina Mozaffari-Jovin^{2

3}, Shima Farrokhi^{1

3}, Maryam Hashemi^{5

6}, Marzieh Lotfi⁷, Majid Ebrahimi Warkiani^{8

9}, Mohammad Reza Abbaszadegan^{10

11}

Affiliations

¹ Student Research Committee, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.
² Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
³ Department of Medical Genetics and Molecular Medicine, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.
⁴ School of Biomedical Engineering, University of Technology Sydney, Sydney, Australia.
⁵ Nanotechnology Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran.
⁶ Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.
⁷ Department of Medical Genetics, School of Medicine, Shahid Sadoughi University of Medical Sciences and Health Services, Yazd, Iran.
⁸ School of Biomedical Engineering, University of Technology Sydney, Sydney, Australia. majid.warkiani@uts.edu.au.
⁹ Institute for Biomedical Materials and Devices (IBMD), Faculty of Science, University of Technology Sydney, Sydney, Australia. majid.warkiani@uts.edu.au.
¹⁰ Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran. abbaszadeganmr@mums.ac.ir.
¹¹ Department of Medical Genetics and Molecular Medicine, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran. abbaszadeganmr@mums.ac.ir.

^# Contributed equally.

Abstract

Rapid advancement in genome editing technologies has provided new promises for treating neoplasia, cardiovascular, neurodegenerative, and monogenic disorders. Recently, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has emerged as a powerful gene editing tool offering advantages, including high editing efficiency and low cost over the conventional approaches. Human pluripotent stem cells (hPSCs), with their great proliferation and differentiation potential into different cell types, have been exploited in stem cell-based therapy. The potential of hPSCs and the capabilities of CRISPR/Cas9 genome editing has been paradigm-shifting in medical genetics for over two decades. Since hPSCs are categorized as hard-to-transfect cells, there is a critical demand to develop an appropriate and effective approach for CRISPR/Cas9 delivery into these cells. This review focuses on various strategies for CRISPR/Cas9 delivery in stem cells.

Keywords: CRISPR/Cas9 system; Delivery technologies; Gene editing; Stem cell therapy; Stem cells.

Publication types

Review

MeSH terms

CRISPR-Cas Systems* / genetics
Cell Differentiation
Gene Editing
Humans
Pluripotent Stem Cells*
Stem Cell Transplantation

Abstract

Publication types

MeSH terms

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