Sharma Bhawna, Chauhan Iti, Kumar Gaurav, Bhardwaj Khushboo, Tiwari Raj Kumar
Department of Pharmaceutics, Dr. K. N. MODI Institute of Pharmaceutical Education and Research, Modinagar, Ghaziabad, UP, India.
Department of Pharmaceutics, I.T.S College of Pharmacy, Muradnagar, Ghaziabad, UP, India.
Microrna. 2024 Oct 31. doi: 10.2174/0122115366319848241022092805.
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.
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病症方面具有巨大潜力。然而，高效递送仍然是一项重大挑战。纳米颗粒就在此发挥作用，成为药物递送领域强大的助力。纳米颗粒能够容纳更大的插入片段，使得更大的Cas9酶和复杂的引导RNA得以整合，从而开启了编辑先前难以触及的基因区域的可能性。其相对简单且可扩展的生产过程使其成为更广泛应用的经济高效选择。值得注意的是，纳米颗粒在体内表现出色，展现出高效的组织穿透和靶向递送能力，这对于在将副作用降至最低的同时最大化治疗效果至关重要。本综述旨在探索基于纳米颗粒的CRISPR/Cas9递送系统的潜力，突出其在基因编辑应用中的优势和挑战。纳米颗粒的种类繁多进一步增强了它们的潜力。例如，聚合物纳米颗粒为CRISPR货物的定制和控释提供了可调节的特性。基于脂质的纳米颗粒促进有效的细胞摄取和内体逃逸，确保CRISPR组件到达目标DNA。甚至以其独特的生物相容性和光热特性而闻名的金纳米颗粒，在光激活编辑策略中也具有前景。非病毒递送系统，尤其是基于纳米颗粒的系统，因其固有优势而脱颖而出。总体而言，证据描绘了一幅充满希望的图景：纳米颗粒不仅是被动载体，而且是CRISPR/Cas9递送领域的积极参与者。它们的多功能性、效率和安全性使其成为未来的关键推动者，在这个未来中，基因编辑可以彻底改变药物开发，为广泛的疾病提供个性化和靶向治疗。
