Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing

Author:

Rostami Neda1,Gomari Mohammad Mahmoudi2,Choupani Edris3,Abkhiz Shadi2,Fadaie Mahmood4,Eslami Seyed Sadegh25,Mahmoudi Zahra2,Zhang Yapei6,Puri Madhu6,Monfared Fatemeh Nafe7,Demireva Elena6,Uversky Vladimir N.8,Smith Bryan Ronain69ORCID,Bencherif Sidi A.101112ORCID

Affiliation:

1. Department of Chemical Engineering Arak University Arak 3848177584 Iran

2. Department of Medical Biotechnology Faculty of Allied Medicine Iran University of Medical Sciences Tehran 1449614535 Iran

3. Department of Molecular Biology and Biochemistry Rutgers University Piscataway NJ 08854 USA

4. Department of Genetics and Molecular Biology School of Medicine Isfahan University of Medical Sciences Isfahan 8174673461 Iran

5. Molecular Proteomics Laboratory Baker Heart and Diabetes Institute Melbourne Victoria 3004 Australia

6. Department of Biomedical Engineering Institute for Quantitative Health Science and Engineering Michigan State University East Lansing MI 48824 USA

7. Department of Virology School of Public Health Tehran University of Medical Sciences Tehran 1416634793 Iran

8. Department of Molecular Medicine and USF Health Byrd Alzheimer's Research Institute Morsani College of Medicine University of South Florida Tampa FL 33612 USA

9. Department of Radiology Stanford University Stanford CA 94305 USA

10. Departments of Chemical Engineering and Bioengineering Northeastern University Boston MA 02115 USA

11. Harvard John A. Paulson School of Engineering and Applied Sciences Harvard University Cambridge MA 02138 USA

12. Biomechanics and Bioengineering Sorbonne University, UTC CNRS UMR 7338, University of Technology of Compiègne 60203 Compiègne France

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.

Funder

National Institutes of Health

National Science Foundation

Publisher

Wiley

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