关键词: Gene therapy Nanostructured drug delivery systems Paediatric Quadrilateral retinoblastoma Retinoblastoma

来  源:   DOI:10.1016/j.heliyon.2024.e32844   PDF(Pubmed)

Abstract:
The most prevalent paediatric vision-threatening medical condition, retinoblastoma (RB), has been a global concern for a long time. Several conventional therapies, such as systemic chemotherapy and focal therapy, have been used for curative purposes; however, the search for tumour eradication with the least impact on surrounding tissues is still ongoing. This review focuses on the genetic origin, classification, conventional treatment modalities, and their combination with nano-scale delivery systems for active tumour targeting. In addition, the review also delves into ongoing clinical trials and patents, as well as emerging therapies such as gene therapy and immunotherapy for the treatment of RB. Understanding the role of genetics in the development of RB has refined its treatment strategy according to the genetic type. New approaches such as nanostructured drug delivery systems, galenic preparations, nutlin-3a, histone deacetylase inhibitors, N-MYC inhibitors, pentoxifylline, immunotherapy, gene therapy, etc. discussed in this review, have the potential to circumvent the limitations of conventional therapies and improve treatment outcomes for RB. In summary, this review highlights the importance and need for novel approaches as alternative therapies that would ultimately displace the shortcomings associated with conventional therapies and reduce the enucleation rate, thereby preserving global vision in the affected paediatric population.
摘要:
最普遍的儿科视力威胁医疗状况,视网膜母细胞瘤(RB),长期以来一直是全球关注的问题。几种常规疗法,如全身化疗和局灶性治疗,已用于治疗目的;然而,寻找对周围组织影响最小的肿瘤根除仍在进行中。这篇综述的重点是遗传起源,分类,常规治疗方式,以及它们与纳米级递送系统的组合,用于主动靶向肿瘤。此外,该审查还深入研究了正在进行的临床试验和专利,以及新兴的疗法,如基因治疗和免疫治疗治疗RB。了解遗传学在RB发育中的作用,已根据遗传类型完善了其治疗策略。新的方法,如纳米结构的药物递送系统,盖伦制剂,nutlin-3a,组蛋白去乙酰化酶抑制剂,N-MYC抑制剂,己酮可可碱,免疫疗法,基因治疗,等。在这次审查中讨论,有可能规避常规疗法的局限性并改善RB的治疗结果。总之,这篇综述强调了作为替代疗法的新方法的重要性和必要性,这些新方法最终将取代与传统疗法相关的缺点,并降低摘除率。从而在受影响的儿科人群中保持全球视野。
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