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Overcoming the Delivery Challenges in CRISPR/Cas9 Gene Editing for Effective Cancer Treatment: A Review of Delivery
Shuting Tang1,2, Xiaoyi Chen3, Xiangmin Tong4
1College of Materials and Engineering, Yangtze Normal University, Chongqing 408100, China.
Abstract:
Therapeutic strategies based on gene editing provide the ability to modify faulty genes contributing to the development of diseases such as cancer by directly altering the cellular machinery. The clustered regularly interspaced short palindromic repeats associated nuclease 9 (CRISPR/Cas9) system is currently the primary tool used for gene editing. Several effective Cas9 variants have already been established to address the complex genetic modifications that arise during diseases. Although gene-editing systems have made significant advancements, a primary obstacle that requires attention is the transportation of CRISPR/Cas to diverse target cells, both in vivo and in vitro, to render them suitable for clinical implementation. Various strategies can be utilized to facilitate the transportation of the CRISPR/Cas systems into mammalian cells. Herein, we reviewed contemporary research about delivery systems for gene-editing systems that interact effectively in biological systems. This review explores the benefits and drawbacks of using extracellular vesicles and viral vectors as vehicles for delivering the CRISPR/Cas system in the context of cancer treatment.
Insights
Gene editing therapies, like CRISPR/Cas9, offer new ways to treat cancer by fixing faulty genes. Efficient delivery of these gene editing tools to target cells remains a key challenge for clinical use.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Gene editing offers therapeutic potential for diseases like cancer by correcting genetic defects.
- The CRISPR/Cas9 system is a leading technology for gene editing, with various Cas9 variants developed for complex genetic modifications.
- Clinical application of gene editing is hindered by challenges in delivering CRISPR/Cas systems to target cells both *in vivo* and *in vitro*.
Purpose of the Study:
- To review current research on delivery systems for gene editing technologies.
- To explore the advantages and disadvantages of different delivery vehicles for CRISPR/Cas systems.
- To discuss the suitability of these delivery systems for clinical implementation in cancer treatment.
Main Methods:
- Literature review of contemporary research on gene editing delivery systems.
- Analysis of extracellular vesicles as delivery vehicles.
- Analysis of viral vectors as delivery vehicles.
Main Results:
- Extracellular vesicles and viral vectors are key strategies for delivering CRISPR/Cas systems into mammalian cells.
- Both approaches have distinct benefits and drawbacks influencing their effectiveness and safety.
- Successful delivery is crucial for the *in vivo* and *in vitro* application of gene editing therapies.
Conclusions:
- Effective delivery systems are essential for translating gene editing technologies into clinical cancer treatments.
- Further research is needed to optimize delivery methods using extracellular vesicles and viral vectors.
- Addressing delivery challenges will accelerate the clinical implementation of CRISPR/Cas-based therapies.
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