Preclinical pharmacology and toxicology study of an AAV8-tATP7B vector for Wilson’s disease
These results establish the efficacy and safety profile of AAV8-tATP7B and provide the rationale for its clinical translation in patients with WD.
Authors
- Mengyuan Wu (ORCID: https://orcid.org/0000-0002-1429-122X)
- Z. Wang (ORCID: https://orcid.org/0009-0002-0815-8673)
- Lei Ye (ORCID: https://orcid.org/0000-0001-9055-1874)
- Fang Liu
- Jian-Gao Fan
- Xuxu Sun
- Wenzheng Zhao
- Mengyu Wang
- Tianyi Ren
- Wuping Li
- Tianwen Lou
Institutions
- Shanghai Jiao Tong University (CN)
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- National Health and Family Planning Commission (CN)
- XinHua Hospital (CN)
- Ruijin Hospital (CN)
- Shanghai Institute of Nutrition and Health (CN)
- Zhejiang Medicine (China) (CN)
- Dali University (CN)
Publication Details
- Journal
- Clinical and Molecular Hepatology
- Published
- 2026-03-04
- DOI
- https://doi.org/10.3350/cmh.2025.1300
- Primary Topic
- Trace Elements in Health
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Shanghai Jiao Tong University
- National Science and Technology Major Project
- School of Medicine, Shanghai Jiao Tong University