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Exploring Non-Viral Methods for the Delivery of Crispr-Cas Ribonucleoprotein to Hematopoietic Stem Cells Publisher Pubmed

Summary: Can CRISPR fix blood disorders? Study finds RNP complexes enhance HSC gene editing safely. #CRISPR #HematologicDisorders

Molaei Z1 ; Jabbarpour Z2 ; Omidkhoda A1 ; Ahmadbeigi N4
Authors

Source: Stem Cell Research and Therapy Published:2024


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. © The Author(s) 2024.
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