视网膜母细胞瘤基因治疗研究进展

Current advance in gene therapy of retinoblastoma

视网膜母细胞瘤（RB）是婴幼儿最常见的眼内恶性肿瘤，传统的治疗方法主要采取眼球或眼眶内容物摘除术、放射疗法及化学疗法，但均不能明显改善预后，而RB的远期影响往往带来局部的破坏性后果。基因疗法为RB的治疗及预后的改善带来了契机，具有传统方法不可比拟的优势。就基因转染载体、基因导人方式、常用的基因技术（如RNA干扰技术）和治疗靶点等方面对RB基因治疗的研究进展进行综述。

Retinoblastoma （RB） is the most common infant eye malignant tumor. Conventional management mainly include enucleation of eyeball,evisceration of orbital tissue, radiotherapy and chemotherapy,but the prognosis is not significantly improved, and its long-term effects are often devastating. Recent years, the development of gene therapy brings a chance for achieving the goal of treatment RB and improving patients＇ prognosis. The progress in gene therapy for RB,the vector of gene transfer,the methods of gene delivery and the commonly used technologies （RNA interference） to the therapeutic targets were reviewed.