Centre for Cellular and Molecular Biology, Hyderabad 500 007, India.
Chem Phys Lipids. 2010 Mar;163(3):245-52. doi: 10.1016/j.chemphyslip.2010.01.001. Epub 2010 Jan 12.
Realization of the potential of nucleic acids as drugs is intricately linked to their in vivo delivery. Cationic lipids demonstrated tremendous potential as safe, efficient and scalable in vitro carriers of nucleic acids. For in vivo delivery of nucleic acids, the extant two component liposomal preparations consisting of cationic lipids and nucleic acids have been largely found to be insufficient. Being a soft matter, liposomes readily respond to many physiological variables leading to complex component and morphological changes, thus confounding the efforts in a priori identification of a "competent" formulation. In the recent past many chemical moieties that provide advantage in facing the challenges of barriers in vivo, were incorporated into cationic lipids to improve the transfection efficiency. The cationic lipids, essential for DNA condensation and protection, definitely require additional components to be efficient in vivo. In addition, formulations of cationic lipid carriers with non-lipidic components, mainly peptides, have demonstrated success in in vivo transfection. The present review describes some recent successes of in vivo nucleic acid delivery by cationic lipids.
核酸类药物的潜力的实现与其体内传递密切相关。阳离子脂质体作为安全、高效和可扩展的核酸体外载体,显示出巨大的潜力。对于核酸的体内传递,现有的由阳离子脂质体和核酸组成的两成分脂质体制剂在很大程度上被发现是不足够的。作为软物质,脂质体容易响应许多生理变量,导致复杂的成分和形态变化,从而使预先确定“有效”制剂的努力变得复杂。在最近的过去,许多在应对体内障碍的挑战方面提供优势的化学部分被掺入阳离子脂质体中,以提高转染效率。对于 DNA 凝聚和保护至关重要的阳离子脂质体,在体内确实需要额外的成分才能有效。此外,带有非脂质成分(主要是肽)的阳离子脂质载体制剂在体内转染方面取得了成功。本综述描述了阳离子脂质体体内核酸传递的一些最新成功案例。