基因治疗的进展已经预示了其治疗涉及遗传功能障碍的广泛疾病的潜力。然而,它的治疗效率和成功的结果高度依赖于理想的基因传递系统的发展。在这个问题上,基于二氧化硅的载体由于其增加的安全性而转移了病毒和其他类型的非病毒载体的一些注意力,易于修改的结构和表面,高稳定性,和成本效益。硅烷化学的多功能性以及二氧化硅与其他材料的结合,如聚合物,脂质,或无机颗粒,导致了具有强大装载能力的载体的发展,有效保护和结合遗传物质的能力,有针对性的交付,和货物的刺激响应释放。使用这些纳米系统作为不同潜在治疗领域的多功能平台,在体外和体内都获得了有希望的结果。比如癌症或脑部治疗,有时结合成像功能。在这里,综述了用于基因治疗的硅基系统的最新进展,包括它们的主要属性,制造方法,表面改性,和潜在的治疗应用。
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.