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Combinatorial cationic lipid-like nanoparticles for efficient intracellular cytotoxic protein delivery

机译:组合阳离子脂质样纳米颗粒,可有效递送细胞内细胞毒性蛋白

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Introduction: Although protein-based drugs have shown success, they have been limited mostly to cytokines, growth factors, enzymes and monoclonal antibodies, all of which function primarily extracellularly. There are a number of diseases, including genetic diseases and cancers, that have the potential to be treated through proteins with an intracellular target. However, proteins alone are not usually able to cross the cell membrane in order to reach their intracellular targets. Hence it is desirable to develop efficient and effective tools as well as strategies that will enable us to deliver therapeutic proteins in their active forms to tumor cells or tissues. Here we present a combinatorial approach for the creation of cationic lipid-like nanoparticles (termed “lipidoids”) to facilitate intracellular cytotoxic protein delivery for the inhibition of tumor cell proliferation.
机译:简介:尽管基于蛋白质的药物已显示出成功,但它们主要限于细胞因子,生长因子,酶和单克隆抗体,所有这些主要在细胞外发挥作用。有许多疾病,包括遗传性疾病和癌症,都有可能通过具有细胞内靶蛋白的蛋白质进行治疗。然而,单独的蛋白质通常不能穿过细胞膜以达到其细胞内靶标。因此,需要开发有效的工具和策略,以使我们能够将其活性形式的治疗性蛋白质递送至肿瘤细胞或组织。在这里,我们提出了一种用于创建阳离子脂质样纳米颗粒(称为“类脂质”)的组合方法,以促进细胞内细胞毒性蛋白的递送,从而抑制肿瘤细胞的增殖。

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