Department of Pharmaceutics, University Institute of Pharmaceutical Sciences, Chandigarh University, Gharuan, Mohali, 140413, India.
Endocr Metab Immune Disord Drug Targets. 2024;24(13):1480-1487. doi: 10.2174/0118715303282573240112104035.
Organelle-specific targeted drug delivery has emerged as a promising approach in the field of drug delivery and therapeutics. This innovative strategy involves the precise delivery of therapeutic agents to specific organelles within cells, such as the nucleus, mitochondria, endoplasmic reticulum, or lysosomes, with the aim of enhancing drug efficacy while minimizing offtarget effects. Despite its tremendous potential, organelle-specific drug delivery faces several key challenges. One major challenge is the development of delivery systems that can accurately navigate the complex intracellular environment and deliver drugs exclusively to the desired organelles. Achieving this level of precision demands advanced nanotechnology and biomaterials engineering. Furthermore, ensuring the safety and biocompatibility of these delivery systems is paramount. Recent advancements in this field include the development of nanocarriers, such as liposomes, nanoparticles, and dendrimers, designed to target specific organelles through ligandreceptor interactions or pH-responsive mechanisms. Additionally, advancements in molecular biology and genetic engineering have enabled the design of genetically encoded organellespecific drug delivery systems. The therapeutic implications of organelle-specific drug delivery are vast. This approach has the potential to revolutionize the treatment of diseases with organelle- specific pathologies, such as neurodegenerative disorders, cancer, and mitochondrial diseases. By precisely targeting the organelles involved in disease progression, the efficacy of therapies can be significantly improved while minimizing collateral damage to healthy tissues.
细胞器特异性靶向药物递送在药物递送和治疗领域已成为一种很有前途的方法。这种创新策略涉及将治疗剂精确递送到细胞内的特定细胞器，如核、线粒体、内质网或溶酶体，旨在提高药物疗效的同时最小化脱靶效应。尽管具有巨大的潜力，但细胞器特异性药物递送面临着几个关键挑战。一个主要的挑战是开发能够准确导航复杂的细胞内环境并将药物专门递送到所需细胞器的输送系统。要实现这种精确性，需要先进的纳米技术和生物材料工程。此外，确保这些输送系统的安全性和生物相容性至关重要。该领域的最新进展包括开发旨在通过配体-受体相互作用或 pH 响应机制靶向特定细胞器的纳米载体，如脂质体、纳米颗粒和树枝状大分子。此外，分子生物学和基因工程的进展使设计具有遗传编码的细胞器特异性药物输送系统成为可能。细胞器特异性药物输送的治疗意义非常广泛。这种方法有可能彻底改变治疗具有细胞器特异性病理的疾病，如神经退行性疾病、癌症和线粒体疾病。通过精确靶向疾病进展涉及的细胞器，可以显著提高治疗效果，同时最小化对健康组织的附带损伤。
