在正常肝脏中，AAV8载体主要转导门脉区的肝细胞，但在脂肪肝肝脏中，这种转导效率发生了改变，AAV5载体反而显示出对中心区肝细胞的优先转导能力。

(MGTX), Wednesday announced that the U.S. Food and Drug Administration has granted the Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber ...