PDF(Pubmed)
Abstract：
The study of gene therapies has been of particular interest in recent decades due to their promising potential to slow or even rescue the degeneration of the retina in inherited retinal dystrophies (IRDs). Here, we review the current approaches to gene therapy trials on IRDs, including the selection of animal models, therapeutic window, vectors and dosages. Mice are typically the first choice of animal models and recombinant adeno-associated virus (rAAV) of serotype 8 is the most common vector for loss-of-function IRDs. Furthermore, the therapeutic window should be considered to ensure efficacy before retinal degeneration occurs if possible, and dosages must be tailored to each approach.
摘要：
近几十年来，基因疗法的研究受到了特别的关注，因为它们具有减缓甚至挽救遗传性视网膜营养不良（IRD）中视网膜变性的潜力。这里,我们回顾了目前对IRD进行基因治疗试验的方法，包括动物模型的选择，治疗窗口，载体和剂量。小鼠通常是动物模型的首选,并且血清型8的重组腺相关病毒(rAAV)是功能丧失IRD的最常见载体。此外，如果可能，应考虑治疗窗口以确保在视网膜变性发生之前有效，剂量必须根据每种方法定制。
