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首页> 外文期刊>The journal of gene medicine >Long-term evaluation of retinal function in Prph2~(Rd2/Rd2) mice following AAV-mediated gene replacement therapy
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Long-term evaluation of retinal function in Prph2~(Rd2/Rd2) mice following AAV-mediated gene replacement therapy

机译：AAV介导的基因替代疗法对Prph2〜（Rd2 / Rd2）小鼠视网膜功能的长期评估

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Background Prph2~(Rd2/Rd2) mice have a retinal degeneration due to a null mutation for the Prph2 gene which encodes a photoreceptor-specific glycoprotein, peripherin2, essential for outer-segment formation. We have previously shown improvement of photoreceptor function at a single time point following AAV-mediated gene replacement therapy. Here we quantify the functional rescue over a 15-week time course and present a detailed analysis of the improvement in retinal function. Methods An AAV2 vector, AAv.rho.rds, carrying a periphering2 c-DNA, was in injected subretinally into 10-day-old Prph2~(Rd2/Rd2) mice. One group was injected at a single time point while in a second group the injections were repeated after 5 days. The effect of treatment was analysed histologically using electron microscopy and electroetinography (ERG) was used to assess functional changes. Treated mice were recorded at regular intervals over 15 weeks. Untreated contralateral eyes served as internal control. Results A significant increase in b-wave amplitude was first noted 3 weeks after treatment of 10-day-old Prph2~(Rd2/Rd2) mice and persisted for up to 14 weeks. An increase in the area of retina exposed to vector resulted in a significant increase in both b-wave amplitude and persistence. Conclusions In this study AAV-mediated gene replacement in Prph2~(Rd2/Rd2) mice resulted in a significant functional improvement over a period of 14 weeks. These results support the utility of gene therapy approaches as treatment for photoreceptor dystrophies.
机译：背景Prph2〜（Rd2 / Rd2）小鼠具有视网膜变性，这是由于Prph2基因的空突变所致，该基因编码外部区域形成所必需的感光细胞特异性糖蛋白，peripherin2。我们先前已经显示了AAV介导的基因替代疗法后单个时间点的光感受器功能的改善。在这里，我们量化了15周的疗程中的功能抢救，并提出了视网膜功能改善的详细分析。方法将携带有周围c-DNA的AAV2载体AAv.rhosrds经视网膜下注射到10日龄的Prph2〜（Rd2 / Rd2）小鼠体内。一组在单个时间点注射，而第二组在5天后重复注射。使用电子显微镜对治疗效果进行组织学分析，并使用电泳图谱（ERG）评估功能变化。在15周内定期记录治疗小鼠。未经处理的对侧眼用作内部对照。结果治疗10日龄的Prph2〜（Rd2 / Rd2）小鼠治疗3周后，首先注意到b波幅度明显增加，并持续长达14周。暴露于载体的视网膜面积增加导致b波振幅和持续性均显着增加。结论在这项研究中，Av介导的Prph2〜（Rd2 / Rd2）小鼠基因替换在14周内导致了显着的功能改善。这些结果支持基因治疗方法作为治疗感光性营养不良的实用性。

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