School of Medical Science & Technology, Indian Institute of Technology Kharagpur, Kharagpur, West Bengal 721302, India.
School of Medical Science & Technology, Indian Institute of Technology Kharagpur, Kharagpur, West Bengal 721302, India.
Colloids Surf B Biointerfaces. 2022 Dec;220:112899. doi: 10.1016/j.colsurfb.2022.112899. Epub 2022 Oct 4.
Over the last two decades, nanoparticulate delivery systems have revolutionized cancer treatment by achieving target-specific delivery, enhanced bioavailability, and improved toxicity profile. The increasing interest in nanotechnology for cancer treatment stems from the unique physicochemical properties of nanoparticles (for instance, small size, surface characteristics, etc.). Indeed, different anticancer drugs can be effectively delivered through nano-delivery systems nowadays. However, the application of such delivery systems in the arena of gene therapy remains in its infancy. Moreover, the treatment of retinoblastoma (RB), an aggressive ocular cancer of childhood, is a major problem in developing countries owing to the late diagnosis of this type of cancer. While adeno-associated virus-based delivery strategies remain the mainstay of the gene delivery method due to their high efficiency, other delivery systems, such as non-viral nanoparticles (NPs) are being developed as alternative therapeutic modalities. Indeed, different nanoparticle formulations such as lipid-based nanoparticles, polymeric nanoparticles, gold nanoparticles have displayed improved gene delivery efficiency in retinal diseases. This review article focuses on the nanoparticle mediated gene therapy approaches in the treatment of RB and highlights the attempts made to develop improved formulations for the treatment of RB. We delineate the current status of NPs as a gene delivery vehicle and cover the future perspective of this exciting field of research. Also, we discuss the achievement, challenges, and opportunities of nanomedicine to treat RB and mention novel engineering approaches that leverage our growing understanding of tumor biology and mechanisms of NPs uptake to develop more effective nanotherapeutics for RB patients.
在过去的二十年中，纳米颗粒给药系统通过实现靶向递药、提高生物利用度和改善毒性特征，彻底改变了癌症治疗。纳米技术在癌症治疗中的应用兴趣日益浓厚，这源于纳米颗粒的独特物理化学性质（例如，小尺寸、表面特性等）。实际上，如今不同的抗癌药物可以通过纳米递药系统有效递送到体内。然而，这种给药系统在基因治疗领域的应用仍处于起步阶段。此外，由于这种癌症的诊断较晚，发展中国家儿童罹患的侵袭性眼部癌症视网膜母细胞瘤（RB）的治疗仍然是一个重大问题。尽管腺相关病毒（adeno-associated virus，AAV）为基础的递送策略因其高效性而仍然是基因递送方法的主要手段，但其他递送系统，如非病毒纳米颗粒（nanoparticles，NPs）也正在被开发作为替代治疗方法。实际上，不同的纳米颗粒制剂，如基于脂质的纳米颗粒、聚合物纳米颗粒、金纳米颗粒，在视网膜疾病中显示出了提高的基因递送效率。本文综述了纳米颗粒介导的基因治疗方法在 RB 治疗中的应用，并强调了为治疗 RB 而开发改进配方的尝试。我们阐述了 NPs 作为基因递送载体的现状，并涵盖了这一令人兴奋的研究领域的未来前景。此外，我们还讨论了纳米医学在治疗 RB 方面的成就、挑战和机遇，并提到了利用我们对肿瘤生物学和 NPs 摄取机制的不断深入了解来开发更有效的纳米疗法的新工程方法。
