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

Autor: Molaei Z; Hematology and blood transfusion science department, School of Allied Medical Sciences, Tehran University of Medical Sciences, Tehran, Iran., Jabbarpour Z; School of Pharmacy & Bioengineering, Guy Hilton Research Centre (GHRC), Keele University, Staffordshire, ST4 7QB, UK., Omidkhoda A; Hematology and blood transfusion science department, School of Allied Medical Sciences, Tehran University of Medical Sciences, Tehran, Iran. a-omidkhoda@tums.ac.ir., Ahmadbeigi N; Gene Therapy Research Center, Digestive Disease Research Institute, Tehran University of Medical Sciences, Tehran, Iran. n-ahmadbeigi@tums.ac.ir.
Jazyk: angličtina
Zdroj: Stem cell research & therapy [Stem Cell Res Ther] 2024 Jul 29; Vol. 15 (1), pp. 233. Date of Electronic Publication: 2024 Jul 29.
DOI: 10.1186/s13287-024-03848-4
Abstrakt: 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.
(© 2024. The Author(s).)
Databáze: MEDLINE
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