Targeted Nanoparticle Delivery of CRISPR−Cas9 System for Precision Genome Editing In vivo

Margherita Fogliano^*
*Correspondence: Margherita Fogliano, Department of Biomedical Technologies, Coburg University of Applied Sciences and Arts, Friedrich-Streib, Coburg, Germany, Email:

Keywords

CRISPR-Cas9 • Genome editing • Nanoparticles • In vivo delivery • Precision medicine • Targeted therapy • Genetic disorders

Introduction

The revolutionary CRISPR-Cas9 genome editing system has brought unprecedented opportunities for precise modifications of the genome. However, the translation of this technology to in vivo applications, especially for therapeutic purposes, necessitates the development of efficient and targeted delivery methods. This article focuses on the integration of nanoparticles as delivery vehicles for the CRISPR-Cas9 system, enabling precise genome editing within living organisms [1].

Literature Review

This section provides a comprehensive exploration of the development and utilization of targeted nanoparticles as an innovative delivery platform for the CRISPR-Cas9 genome editing system, particularly focusing on its in vivo applications:

Nanoparticle design and optimization: The foundation of efficient genome editing in vivo lies in the meticulous design and optimization of nanoparticles. We delve into the selection of nanoparticle materials, emphasizing their biocompatibility, stability, and capacity to encapsulate and protect the CRISPR-Cas9 cargo. Furthermore, size, surface charge, and surface modifications are discussed as critical factors influencing cellular uptake and controlled release [2].

Targeted delivery strategies: Achieving precise cell-type or tissuespecific delivery is a paramount challenge. We explore advanced strategies that endow nanoparticles with the ability to home in on specific targets. Surface functionalization with ligands, antibodies, or aptamers is examined in detail, demonstrating how these modifications enhance the specificity and affinity of nanoparticles for particular cell populations. This targeted approach not only maximizes the therapeutic effect but also minimizes off-target genome modifications, a critical concern in genome editing applications.

In vivo applications: The heart of this section lies in the examination of recent in vivo studies that have harnessed nanoparticle-based delivery systems for precision genome editing. We discuss these studies in the context of addressing genetic disorders, emphasizing the potential therapeutic benefits realized through this technology. Specific cases, such as the correction of mutations responsible for monogenic diseases or the modulation of genes associated with complex conditions, exemplify the versatility and effectiveness of this approach [3].

Safety considerations: While the promise of precision genome editing is tremendous, ensuring the safety of in vivo applications is of paramount importance. The discussion touches on potential immunogenicity, cytotoxicity, and long-term effects associated with nanoparticle-based delivery. It emphasizes the necessity of rigorous safety assessments and monitoring strategies to mitigate any adverse outcomes [4].

Future directions: In the ever-evolving landscape of CRISPR-based genome editing, the integration of nanoparticles opens new possibilities. We explore future directions, including the potential for synergy between nanoparticle delivery and emerging CRISPR technologies such as base editing and prime editing. These technologies may further enhance the precision and scope of genome modifications while addressing some of the limitations associated with traditional CRISPR-Cas9 systems.

This comprehensive exploration of nanoparticle-mediated delivery for the CRISPR-Cas9 system underscores its transformative potential in the field of precision genome editing in vivo. As technology continues to advance, the synergy between nanotechnology and genome editing promises to reshape the landscape of personalized medicine, gene therapy, and the treatment of genetic disorders, offering new hope for patients with previously untreatable conditions [5].

Discussion

The Discussion section critically examines the advantages and challenges associated with using nanoparticles for CRISPR-Cas9 delivery in vivo. We address issues of immunogenicity, toxicity, and off-target effects, as well as the need for long-term safety assessments. Furthermore, we explore the potential synergy between nanoparticle delivery and emerging CRISPR technologies, such as base editing and prime editing [6].

Conclusion

In conclusion, the integration of targeted nanoparticles with the CRISPRCas9 system offers a promising avenue for precision genome editing in vivo. This approach holds great potential for the treatment of genetic disorders, with the capability to address specific cell populations and tissues while minimizing unintended genetic modifications. As nanoparticle design and delivery techniques continue to advance, the future of genome editing as a therapeutic tool appears increasingly promising, ushering in an era of precision medicine and personalized genetic therapies.

Acknowledgement

None.

Conflict of Interest

None.

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