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Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications

机译:基于二氧化硅的基因递送系统:从设计到治疗应用

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摘要

Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, silica-based vectors have diverted some attention from viral and other types of non-viral vectors due to their increased safety, easily modifiable structure and surface, high stability, and cost-effectiveness. The versatility of silane chemistry and the combination of silica with other materials, such as polymers, lipids, or inorganic particles, has resulted in the development of carriers with great loading capacities, ability to effectively protect and bind genetic material, targeted delivery, and stimuli-responsive release of cargos. Promising results have been obtained both in vitro and in vivo using these nanosystems as multifunctional platforms in different potential therapeutic areas, such as cancer or brain therapies, sometimes combined with imaging functions. Herein, the current advances in silica-based systems designed for gene therapy are reviewed, including their main properties, fabrication methods, surface modifications, and potential therapeutic applications.
机译:基因治疗的进步一直预示着它对治疗涉及遗传遗役的广泛疾病的潜力。然而,其治疗效率和成功结果高度依赖于理想基因递送系统的发展。就此而言,基于二氧化硅的载体由于其增加的安全性,易于修饰的结构和表面,高稳定性和成本效益而转移了病毒和其他类型的非病毒载体。硅烷化学的多功能性和二氧化硅与其他材料的组合,例如聚合物,脂质或无机颗粒,导致载体的发育具有很大的装载能力,有效保护和结合遗传物质,靶向递送和刺激的能力 - 宣传卡尔科斯。有希望的结果已经在体外和体内使用这些纳米系统作为多功能平台,例如不同潜在的治疗区域,例如癌症或脑疗法,有时与成像功能相结合。这里,综述了设计用于基因疗法的基于二氧化硅的系统的电流进展,包括它们的主要性质,制造方法,表面改性和潜在的治疗应用。

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