This review article discusses the therapeutic potential of gene editing, focusing on mRNA-based delivery systems. It highlights the advancements in gene editing technologies like CRISPR-Cas systems and base editors, and examines their applications in treating genetic disorders.
Key Points
1. Gene Editing Technologies:
- CRISPR-Cas Systems: Recognize target DNA sequences via RNA-DNA hybridization, enabling precise cuts in the DNA.
- Base Editors: Allow single-nucleotide changes without creating double-stranded breaks, reducing the risk of off-target effects.
2. mRNA Delivery Advantages:
- Transient expression provides controlled, time-limited therapeutic effects.
- Avoids genomic integration, preserving the host genome’s integrity.
- Reduced immunogenicity compared to viral vectors.
3. Applications:
- Ex Vivo Gene Editing: Genetic modification of cells outside the body before reintroduction.
- In Vivo Gene Editing: Direct editing of genes within the body.
4. Next-Generation CRISPR Systems:
- Designed to minimize off-target effects and enable precise gene modifications.
- Include base editors capable of making specific nucleotide changes without DNA cleavage.
5. Therapeutic Potential:
- mRNA-based gene editing shows promise for treating a variety of genetic disorders.
- Focus on improving delivery methods to enhance therapeutic efficacy and safety.
Conclusion
The combination of CRISPR technology and mRNA delivery represents a significant advancement in gene therapy. This approach offers a safer and more precise method for gene editing, with potential applications in treating a wide range of genetic diseases. Ongoing research aims to further refine these technologies and explore new therapeutic possibilities.
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Gene editing therapeutics based on mRNA delivery, Advanced Drug Delivery Reviews, Volume 200, 2023, 115026, ISSN 0169-409X, https://doi.org/10.1016/j.addr.2023.115026.
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