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

Author:

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

Abstract

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. Graphical Abstract

Funder

Australian Research Council

Cancer Institute NSW

University of Technology Sydney

Publisher

Springer Science and Business Media LLC

Subject

General Medicine

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