【評論主題】41.先天性夜盲症之基因療法產品 voretigene neparvovec-rzyl，所使用之基因載體為何？(A)clustered regularly interspaced short pali

【評論內容】

Luxturna （voretigene neparvovec-rzyl )works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.

參考網址：https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss