Effective AAV-mediated gene replacement therapy in retinal organoids modelling AIPL1-associated LCA4

Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristán-Reviriego, P. Perdigão, Amy Leung, J. van der Spuy
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在模拟 AIPL1 相关 LCA4 的视网膜器官组织中进行有效的 AAV 介导的基因替代治疗
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