Delivery of CRISPR/Cas9 for Genome Editing: Review Abstract Articles
Delivery of CRISPR/Cas9 for Genome Editing: Review Abstract Articles
sadaf safaei,1,*Hamid Mir Mohammad Sadeghi,2Mina Mirian,3
1. Department of pharmaceutical biotechnology, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran 2. Department of pharmaceutical biotechnology, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran 3. Department of pharmaceutical biotechnology, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran
Introduction: In recent years, genome engineering technologies based on the CRISPR-associated RNA-guided endonuclease Cas9 has provided the ability to rapidly and economically introduce sequence-specific modifications depends on the generation of double-strand break (DSB) and DNA repair process into the genomes of cell and organisms. CRISPR/Cas system is the most flexible and user-friendly platform for genome editing. Gene modification can be introduced into the animal genome through homologous recombination and embryonic stem cell technology. Genetically modified animals, especially gene knockout editing is one of great interest in the prevention and treatment of human diseases. Safe and efficient delivery of CRISPR/Cas9 systems is still a challenge. In this review, we discuss CRISPR/Cas9 delivery methods for Genome Editing.
Methods: CRISPR/Cas system is the most flexible and user-friendly platform for genome editing. Safe and efficient delivery of CRISPR/Cas9 systems is still a challenge. Non-viral vectors, viral vectors, and physical delivery are the most widely used method for delivery of CRISPR/Cas9 for genome editing.
Results: Viral vectors are efficient in gene delivery, but they have some contraindication due to many drawbacks such as off-target effect, immunogenic and inflammatory responses, limited packaging capacity, and high cost in production.
Non-viral vectors, including Nano carriers and nanoparticles such as Nano polymeric- and lipid-based structures, rigid nanoparticles, nanoparticles coupled to specific ligand systems including arginine–glycine–aspartate (RGD) peptide, porous silicon, mesoporous silica, metal–organic, cell-penetrating peptides.
The physical delivery, including microinjection, electroporation and hydrodynamic delivery show high efficiency for the application in vitro, but not satisfy for in vivo application.
Conclusion: CRISPR/Cas9 technology has been used to generate target genome modifications. Genetically modified animals, especially gene knockout editing is one of great interest in the prevention and treatment of human diseases. Non-viral vectors based on Nano carriers plays an important role for targeting delivery of CRISPR/Cas9 systems due to increase the circulation time, low toxicity, biocompatibility, and facilitating scaled up.
Nano carrier-based delivery systems suggesting that they can be potential promising platform for Safe and efficient delivery of CRISPR/Cas9 systems.