Luxturna (voretigene neparvovec-rzyl) is an adenovirus-associated viral vector serotype 2 gene therapy containing the human RPE65 gene for treatment of patients with vision loss due to RPE65-mediated inherited retinal disease. Gene therapy utilizes a viral vector to carry the desired genetic information to target cells; vectors that are successfully transduced into target cells utilize the cell to express the proteins of interest. The goal of gene therapy is to provide a sustained therapeutic benefit via continual expression of the proteins that modulate the pathogenesis of the relevant disease. Luxturna is injected subretinally under general anesthesia.
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