Xia Jianhua, Gu Lei, Pan Qing
The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.
Front Ophthalmol (Lausanne). 2023 Jun 27;3:1193595. doi: 10.3389/fopht.2023.1193595. eCollection 2023.
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.
近几十年来，基因疗法的研究备受关注，因为它们有望减缓甚至挽救遗传性视网膜营养不良（IRD）中视网膜的退化。在此，我们综述了目前针对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，8型重组腺相关病毒（rAAV）是功能丧失性IRD最常用的载体。此外，应考虑治疗窗口，尽可能在视网膜退化发生之前确保疗效，并且剂量必须根据每种方法进行调整。
