Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells.
Stem Cell Res Ther
; 15(1): 233, 2024 Jul 29.
Article
in En
| MEDLINE
| ID: mdl-39075609
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.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Ribonucleoproteins
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Hematopoietic Stem Cells
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CRISPR-Cas Systems
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Gene Editing
Limits:
Animals
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Humans
Language:
En
Journal:
Stem Cell Res Ther
Year:
2024
Document type:
Article
Affiliation country:
Country of publication: