纳米粒子载体:精确 CRISPR/Cas9 基因编辑的新时代。

Bhawna Sharma, Iti Chauhan, Gaurav Kumar, Khushboo Bhardwaj, Raj Kumar Tiwari
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引用次数: 0

摘要

革命性的 CRISPR/Cas9 基因编辑技术在治疗遗传疾病和应对癌症等疾病方面具有巨大潜力。然而,高效给药仍然是一项重大挑战。这就是纳米颗粒发挥作用的地方,它是药物递送领域的强大盟友。纳米颗粒可以容纳更大的插入尺寸,从而能够加入更大的 Cas9 酶和复杂的引导 RNA,为编辑以前无法访问的基因区域提供了可能。它们的生产工艺相对简单且可扩展,使其在更广泛的应用中具有成本效益。值得注意的是,纳米颗粒在体内表现出色,具有高效的组织穿透性和靶向递送能力,这对于最大限度地提高治疗效果并减少副作用至关重要。本综述旨在探讨基于纳米颗粒的 CRISPR/Cas9 给药系统的潜力,突出它们在基因编辑应用中的优势和挑战。纳米颗粒的多样性进一步增强了它们的潜力。例如,聚合物纳米颗粒具有可调特性,可实现 CRISPR 货物的定制和可控释放。基于脂质的纳米颗粒可促进细胞的有效吸收和内泌体逃逸,确保 CRISPR 成分到达目标 DNA。即使是以其独特的生物相容性和光热特性而闻名的金纳米粒子,也有望用于光激活编辑策略。非病毒递送系统,尤其是基于纳米颗粒的系统,因其固有的优势而脱颖而出。总之,这些证据描绘了一幅充满希望的图景:纳米颗粒不仅仅是被动的载体,而是 CRISPR/Cas9 传输领域的积极参与者。它们的多功能性、高效性和安全性使其成为未来基因编辑彻底改变药物开发的关键推动力,为各种疾病提供个性化的靶向疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Nanoparticle Carriers: A New Era of Precise CRISPR/Cas9 Gene Editing.

The revolutionary CRISPR/Cas9 gene editing technology holds immense potential for treating genetic diseases and tackling conditions like cancer. However, efficient delivery remains a significant challenge. This is where nanoparticles come into play, emerging as powerful allies in the realm of drug delivery. Nanoparticles can accommodate larger insertion sizes, enabling the incorporation of larger Cas9 enzymes and complex guide RNAs, thus opening up the possibility of editing previously inaccessible genetic regions. Their relatively straightforward and scalable production processes make them cost-effective options for wider applications. Notably, nanoparticles excel in vivo, demonstrating efficient tissue penetration and targeted delivery, which are crucial for maximizing therapeutic impact while minimizing side effects. This review aims to explore the potential of nanoparticle-based delivery systems for CRISPR/Cas9, highlighting their advantages and challenges in gene editing applications. The diverse range of nanoparticles further bolsters their potential. Polymeric nanoparticles, for instance, offer tunable properties for customization and controlled release of the CRISPR cargo. Lipid-based nanoparticles facilitate efficient cellular uptake and endosomal escape, ensuring the CRISPR components reach the target DNA. Even gold nanoparticles, known for their unique biocompatibility and photothermal properties, hold promise in light-activated editing strategies. Non-viral delivery systems, particularly those based on nanoparticles, stand out due to their inherent advantages. Collectively, the evidence paints a promising picture: nanoparticles are not merely passive carriers but active participants in the CRISPR/Cas9 delivery landscape. Their versatility, efficiency, and safety position them as key enablers of a future where gene editing can revolutionize drug development, offering personalized and targeted therapies for a wide range of diseases.

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