Cardoso Raiane Vieira, Pereira Patricia Ribeiro, Freitas Cyntia Silva, Paschoalin Vania Margaret Flosi
Programa de Pós-Graduação em Ciência de Alimentos e Programa de Pós-Graduação em Quimica, Instituto de Química, Universidade Federal do Rio de Janeiro, Av. Athos da Silveira Ramos 149-sala 545-Cidade Universitária, Rio de Janeiro 21941-909, RJ, Brazil.
Pharmaceutics. 2022 Dec 15;14(12):2808. doi: 10.3390/pharmaceutics14122808.
Drug delivery systems are believed to increase pharmaceutical efficacy and the therapeutic index by protecting and stabilizing bioactive molecules, such as protein and peptides, against body fluids' enzymes and/or unsuitable physicochemical conditions while preserving the surrounding healthy tissues from toxicity. Liposomes are biocompatible and biodegradable and do not cause immunogenicity following intravenous or topical administration. Still, their most important characteristic is the ability to load any drug or complex molecule uncommitted to its hydrophobic or hydrophilic character. Selecting lipid components, ratios and thermo-sensitivity is critical to achieve a suitable nano-liposomal formulation. Nano-liposomal surfaces can be tailored to interact successfully with target cells, avoiding undesirable associations with plasma proteins and enhancing their half-life in the bloodstream. Macropinocytosis-dynamin-independent, cell-membrane-cholesterol-dependent processes, clathrin, and caveolae-independent mechanisms are involved in liposome internalization and trafficking within target cells to deliver the loaded drugs to modulate cell function. A successful translation from animal studies to clinical trials is still an important challenge surrounding the approval of new nano-liposomal drugs that have been the focus of investigations. Precision medicine based on the design of functionalized nano-delivery systems bearing highly specific molecules to drive therapies is a promising strategy to treat degenerative diseases.
药物递送系统被认为可通过保护和稳定生物活性分子（如蛋白质和肽），使其免受体液中的酶和/或不合适的物理化学条件影响，同时保护周围健康组织免受毒性，从而提高药物疗效和治疗指数。脂质体具有生物相容性和可生物降解性，静脉内或局部给药后不会引起免疫原性。然而，它们最重要的特性是能够装载任何药物或复杂分子，而不受其疏水或亲水特性的限制。选择脂质成分、比例和热敏感性对于获得合适的纳米脂质体制剂至关重要。纳米脂质体表面可以进行定制，以成功地与靶细胞相互作用，避免与血浆蛋白发生不良结合，并延长其在血液中的半衰期。巨胞饮作用（不依赖发动蛋白、依赖细胞膜胆固醇的过程）、网格蛋白和小窝蛋白非依赖机制参与脂质体在靶细胞内的内化和运输，以递送负载的药物来调节细胞功能。从动物研究成功转化到临床试验仍然是围绕新型纳米脂质体药物批准的一项重要挑战，这些药物一直是研究的重点。基于设计带有高度特异性分子以驱动治疗的功能化纳米递送系统的精准医学是治疗退行性疾病的一种有前景的策略。
