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Exploring Advanced Crispr Delivery Technologies for Therapeutic Genome Editing Publisher



Rostami N1 ; Gomari MM2 ; Choupani E3 ; Abkhiz S2 ; Fadaie M4 ; Eslami SS2, 5 ; Mahmoudi Z2 ; Zhang Y6 ; Puri M6 ; Monfared FN7 ; Demireva E6 ; Uversky VN8 ; Smith BR6, 9 ; Bencherif SA10, 11, 12
Authors
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Authors Affiliations
  1. 1. Department of Chemical Engineering, Arak University, Arak, 3848177584, Iran
  2. 2. Department of Medical Biotechnology, Faculty of Allied Medicine, Iran University of Medical Sciences, Tehran, 1449614535, Iran
  3. 3. Department of Molecular Biology and Biochemistry, Rutgers University, Piscataway, 08854, NJ, United States
  4. 4. Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, 8174673461, Iran
  5. 5. Molecular Proteomics Laboratory, Baker Heart and Diabetes Institute, Melbourne, 3004, VIC, Australia
  6. 6. Department of Biomedical Engineering, Institute for Quantitative Health Science and Engineering, Michigan State University, East Lansing, 48824, MI, United States
  7. 7. Department of Virology, School of Public Health, Tehran University of Medical Sciences, Tehran, 1416634793, Iran
  8. 8. Department of Molecular Medicine and USF Health Byrd Alzheimer's Research Institute, Morsani College of Medicine, University of South Florida, Tampa, 33612, FL, United States
  9. 9. Department of Radiology, Stanford University, Stanford, 94305, CA, United States
  10. 10. Departments of Chemical Engineering and Bioengineering, Northeastern University, Boston, 02115, MA, United States
  11. 11. Harvard John A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, 02138, MA, United States
  12. 12. Biomechanics and Bioengineering, Sorbonne University, UTC CNRS UMR 7338, University of Technology of Compiegne, Compiegne, 60203, France

Source: Small Science Published:2024


Abstract

The genetic material within cells plays a pivotal role in shaping the structure and function of living organisms. Manipulating an organism's genome to correct inherited abnormalities or introduce new traits holds great promise. Genetic engineering techniques offers promising pathways for precisely altering cellular genetics. Among these methodologies, clustered regularly interspaced short palindromic repeat (CRISPR), honored with the 2020 Nobel Prize in Chemistry, has garnered significant attention for its precision in editing genomes. However, the CRISPR system faces challenges when applied in vivo, including low delivery efficiency, off-target effects, and instability. To address these challenges, innovative technologies for targeted and precise delivery of CRISPR have emerged. Engineered carrier platforms represent a substantial advancement, improving stability, precision, and reducing the side effects associated with genome editing. These platforms facilitate efficient local and systemic genome engineering of various tissues and cells, including immune cells. This review explores recent advances, benefits, and challenges of CRISPR-based genome editing delivery. It examines various carriers including nanocarriers (polymeric, lipid-derived, metallic, and bionanoparticles), viral particles, virus-like particles, and exosomes, providing insights into their clinical utility and future prospects. © 2024 The Author(s). Small Science published by Wiley-VCH GmbH.
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