视网膜母细胞瘤是儿童最常见的眼内恶性肿瘤。由于延迟诊断，这种罕见疾病在发展中国家的治疗仍具有挑战性。目前的疗法主要包括手术、放疗和化疗。辐射和化疗药物的副作用已被报道为高死亡率和影响患者生活质量的原因之一。由化疗药物分布至非癌细胞而引发的全身副作用是巨大的，并被认为是大多数有效的抗癌药物在临床试验中失败的原因之一。纳米传递系统具有靶向传递、增强渗透和滞留效果、增加生物利用度以及改善毒性剖面的潜力，有可能彻底改变癌症治疗。尽管关于纳米颗粒在视网膜母细胞瘤中的益处的丰富证据，但这种载体的临床转化仍然没有得到必要的关注。本文回顾了视网膜母细胞瘤的当前和新兴治疗选择，重点关注最近针对使用不同类别的纳米颗粒诊断和治疗视网膜母细胞瘤的研究。同时介绍了配体结合和智能纳米颗粒在将抗癌和成像药物主动靶向到肿瘤细胞上的使用。此外，本综述讨论了将这种纳米载体转化为视网膜母细胞瘤治疗临床应用的前景和挑战。本综述可能为制剂科学家提供新的思路，以促进为患有视网膜母细胞瘤的儿童开发更有效和更安全的药物。本期刊由皇家化学学会拥有版权。

Retinoblastoma is the most common intraocular malignancy in children. The treatment of this rare disease is still challenging in developing countries due to delayed diagnosis. The current therapies comprise mainly surgery, radiotherapy and chemotherapy. The adverse effects of radiation and chemotherapeutic drugs have been reported to contribute to the high mortality rate and affect patients' quality of life. The systemic side effects resulting from the distribution of chemotherapeutic drugs to non-cancerous cells are enormous and have been recognized as one of the reasons why most potent anticancer compounds fail in clinical trials. Nanoparticulate delivery systems have the potential to revolutionize cancer treatment by offering targeted delivery, enhanced penetration and retention effects, increased bioavailability, and an improved toxicity profile. Notwithstanding the plethora of evidence on the beneficial effects of nanoparticles in retinoblastoma, the clinical translation of this carrier is yet to be given the needed attention. This paper reviews the current and emerging treatment options for retinoblastoma, with emphasis on recent investigations on the use of various classes of nanoparticles in diagnosing and treating retinoblastoma. It also presents the use of ligand-conjugated and smart nanoparticles in the active targeting of anticancer and imaging agents to the tumour cells. In addition, this review discusses the prospects and challenges in translating this nanocarrier into clinical use for retinoblastoma therapy. This review may provide new insight for formulation scientists to explore in order to facilitate the development of more effective and safer medicines for children suffering from retinoblastoma.This journal is © The Royal Society of Chemistry.