腺相关病毒(AAV)是一种常用的体内基因递送载体,是基因治疗的“黄金载体”。截至2025年1月,全球已有8款AAV基因疗法获批上市,219项AAV相关疗法正处于临床研究中(数据来源于医药魔方NextPharma),发展势头迅猛。然而,AAV治疗也面临 ...
包括携带 TGFβ2 的 AAV - IKV - TGFβ2 CS 、表达 decorin 的 AAV - IKV - Decorin 和表达绿色荧光蛋白(GFP)的 AAV - IKV - GFP 等;其次,通过对小鼠和非人灵长类动物(NHP)进行前房注射,实现基因递送;然后利用组织处理、免疫染色、眼压测量等实验技术,对实验动物的眼部 ...
"We used GFP and AAV as a proof of concept to show that this is a method that works on data sets that are very ...
This pipeline resulted in several viral variants, but Deverman and his team decided to advance the most successful variant, which they called AAV-PHP.B, for a head-to-head comparison with AAV9. For ...
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