Article
Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy.
Gene therapy - 1 Aug 2020
Qu Bin, Wu Shijing, Jiao Guanyi, Zou Xuan, Li Zhikun, Guo Lu, Sun Xuehan, Huang Cheng, Sun Zixi, Zhang Ying, Li Hui, Zhou Qi, Sui Ruifang, Li Wei
Abstract excerpt
Lipid metabolic deficiencies are associated with many genetic disorders. Bietti crystalline dystrophy (BCD), a blindness-causing inherited disorder with changed lipid profiles, is more common in Chinese and Japanese than other populations. Our results reveal that mouse models lacking Cyp4v3 have less physiological and functional changes than those of BCD patients with this gene defect. After the administration of...
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