Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells

Molaei, Zahra; Jabbarpour, Zahra; Omidkhoda, Azadeh; Ahmadbeigi, Naser

Abstract

Gene manipulation of hematopoietic stem cells (HSCs) using the CRISPR/Cas system as a potent genome editing tool holds immense promise for addressing hematologic disorders. An essential hurdle in advancing this treatment lies in effectively delivering CRISPR/Cas to HSCs. While various delivery formats exist, Ribonucleoprotein complex (RNP) emerges as a particularly efficient option. RNP complexes offer enhanced gene editing capabilities, devoid of viral vectors, with rapid activity and minimized off-target effects. Nevertheless, novel delivery methods such as microfluidic-based techniques, filtroporation, nanoparticles, and cell-penetrating peptides are continually evolving. This study aims to provide a comprehensive review of these methods and the recent research on delivery approaches of RNP complexes to HSCs.

Citation

Molaei, Z., Jabbarpour, Z., Omidkhoda, A., & Ahmadbeigi, N. (in press). Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells. Stem Cell Research and Therapy, 15(1), Article 233. https://doi.org/10.1186/s13287-024-03848-4

• Journal Article Type: Review
• Acceptance Date: Jul 12, 2024
• Online Publication Date: Jul 29, 2024
• Deposit Date: Aug 5, 2024
• Journal: Stem Cell Research & Therapy
• Electronic ISSN: 1876-7753
• Publisher: Springer Verlag
• Peer Reviewed: Peer Reviewed
• Volume: 15
• Issue: 1
• Article Number: 233
• DOI: https://doi.org/10.1186/s13287-024-03848-4
• Keywords: Genome editing, Hematopoietic stem cell, CRISPR-Cas System, Delivery strategies, Genetic therapy
• Public URL: https://keele-repository.worktribe.com/output/881954