Shuang Luo, Hao Jiang, Qingwei Li, Yingfei Qin, Shiping Yang, Jing Li, Lingli Xu, Yan Gou, Yafei Zhang, Fengjiang Liu, Xiao Ke, Qiang Zheng, Xun Sun
Recombinant adeno-associated viruses (rAAVs) have emerged as promising gene therapy vectors due to their proven efficacy and safety in clinical applications. In non-human primates (NHPs), rAAVs are administered via suprachoroidal injection at a higher dose. However, high doses of rAAVs tend to increase additional safety risks. Here, we present a novel AAV capsid (AAVv128), which exhibits significantly enhanced transduction efficiency for photoreceptors and retinal pigment epithelial (RPE) cells, along with a broader distribution across the layers of retinal tissues in different animal models (mice, rabbits, and NHPs) following intraocular injection...
May 6, 2024: Nature Communications