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



Molaei Z1 ; Jabbarpour Z2 ; Omidkhoda A1 ; Ahmadbeigi N4
Authors
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Authors Affiliations
  1. 1. Hematology and blood transfusion science department, School of Allied Medical Sciences, Tehran University of Medical Sciences, Tehran, Iran
  2. 2. School of Pharmacy & amp
  3. 3. Bioengineering, Guy Hilton Research Centre (GHRC), Keele University, Staffordshire, ST4 7QB, United Kingdom
  4. 4. Gene Therapy Research Center, Digestive Disease Research Institute, Tehran University of Medical Sciences, Tehran, Iran

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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