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生物材料介导的CRISPR/Cas9递送:基因治疗中的近期挑战与机遇

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy.

作者信息

Dubey Ankit Kumar, Mostafavi Ebrahim

机构信息

Global Research and Publishing Foundation, New Delhi, India.

Institute of Scholars, Bengaluru, Karnataka, India.

出版信息

Front Chem. 2023 Sep 28;11:1259435. doi: 10.3389/fchem.2023.1259435. eCollection 2023.

Abstract

The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. Biomaterials play a vital role in shielding CRISPR/Cas9 components, such as lipid nanoparticles or viral vectors, from immunological processes and degradation, extending their effectiveness. By utilizing the flexibility of biomaterials, tailored systems can be designed to address specific genetic diseases, paving the way for personalized therapeutics. Furthermore, this delivery method offers promising avenues in combating viral illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate site-specific gene modifications, ensuring effective delivery to infected cells while minimizing off-target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target effects, ensuring long-term safety, and establishing scalable production techniques. Thorough research, pre-clinical investigations, and rigorous safety evaluations are imperative for successful translation from the laboratory to clinical applications. In this review, we discussed how CRISPR/Cas9 delivery using biomaterials revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities with the potential to significantly improve human health and quality of life.

摘要

在传染病基因治疗中使用生物材料递送CRISPR/Cas9具有巨大潜力。这种创新方法将CRISPR/Cas9的优势与生物材料的保护特性相结合,既能实现精确高效的基因编辑,又能提高安全性。生物材料在保护CRISPR/Cas9组件(如脂质纳米颗粒或病毒载体)免受免疫过程和降解影响方面发挥着至关重要的作用,从而延长其有效性。通过利用生物材料的灵活性,可以设计出定制系统来解决特定的遗传疾病,为个性化治疗铺平道路。此外,这种递送方法通过精确修饰病原体基因组并降低其致病性,为对抗病毒性疾病提供了有前景的途径。生物材料有助于实现位点特异性基因修饰,确保有效递送至感染细胞,同时将脱靶效应降至最低。然而,挑战依然存在,包括优化递送效率、减少脱靶效应、确保长期安全性以及建立可扩展的生产技术。从实验室成功转化到临床应用,深入的研究、临床前调查和严格的安全性评估必不可少。在本综述中,我们讨论了使用生物材料递送CRISPR/Cas9如何彻底改变基因治疗和传染病治疗,提供精确且安全的编辑能力,有可能显著改善人类健康和生活质量。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5911/10568484/dfab15eea4e9/fchem-11-1259435-g001.jpg

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