Article
Long-Term Correction of Copper Metabolism in Wilson's Disease Mice with AAV8 Vector Delivering Truncated ATP7B.
Human gene therapy - 1 Dec 2019
Leng Yingying, Li Ping, Zhou Lifang, Xiao Lin, Liu Yu, Zheng Zhaoyue, Qin Fengming, Hao Qiukui, Xu Heng, Yao Shaohua, Dong Biao
Abstract excerpt
Wilson's disease (WD) is an autosomal recessive disorder of copper metabolism caused by mutations in the ATP7B gene encoding a liver active copper transport enzyme. Gene therapy with adeno-associated virus (AAV) carrying full-length ATP7B, which is about 4.4 kb, was shown to rescue copper metabolism disorder in WD mouse model. However, due to its relatively large size, the AAV vector containing full-length ATP7B...
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