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Ningdi Luo

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Articles

Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial

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By Mengyue Niu, Jifeng Guo, Yuchao Yang, qian Xu, Haiyan Zhou, Pingchen Zhang, Yuanyuan Li, Ningyuan Wang, Chunyi Wang, Dianyou Li, Zhengyu Lin, Peng Huang, Zhiquan Yang, Yanyan Song, Jing Zheng, Wei Jiang, Zengmin Du, Huirong Peng, Yuyan Tan, Xintian Hu, Xiao Xiao, Lu Shen, Jiaji Lin, Jun Liu, Ningdi Luo, Zhuanyi Yang, Yuanxin Qi
| Nature Verified
Abstract Restoring striatal dopamine synthesis is a promising gene therapy strategy for Parkinson’s disease. Previous adeno-associated virus-mediated aromatic L-amino acid decarboxylase (AADC) monotherapies remain dependent on exogenous levodopa, whereas multigene delivery is constrained by strict adeno-associated virus packaging limits. A ‘dual approach’ targeting the two rate-limiting enzymes, tyrosine hydroxylase (TH) and AADC, offers the potential for autonomous dopamine synthesis.
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