https://www.selleckchem.com/pr....oducts/nuciferine.ht
In conclusion, SACF and GILA are both attractive and valuable additions to preclinical safety assessment of gene therapy products.Patients with Zellweger spectrum disorder (ZSD) commonly present with vision loss due to mutations in PEX genes required for peroxisome assembly and function. Here, we evaluate PEX1 retinal gene augmentation therapy in a mouse model of mild ZSD bearing the murine equivalent (PEX1-p[Gly844Asp]) of the most common human mutation. Experimental adeno-associated virus 8.cytomegalovirus.human PEX1.hemagglutinin
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