Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates
The preclinical evaluation confirms that AAV9-coSMN1 is a safe and effective therapeutic candidate for SMA, with potential applicability across various phenotypes. The study provides critical data supporting its advancement to clinical trials, underscoring its promise for broader neurological applications.
Authors
- Tianyi Zhao
- Wu Zhi (ORCID: https://orcid.org/0000-0002-8160-6615)
- Yixian Zhang (ORCID: https://orcid.org/0000-0002-6429-3127)
- Zheyue Dong
- Jing Liang Qin (ORCID: https://orcid.org/0000-0002-8302-0968)
- Tian Xue (ORCID: https://orcid.org/0000-0001-6187-122X)
- Lina Zhang (ORCID: https://orcid.org/0000-0002-2018-1756)
- Li Li (ORCID: https://orcid.org/0000-0002-1293-0470)
- Sheng Wang (ORCID: https://orcid.org/0000-0003-4041-3216)
- Xiaoyan Dong
- Jun He (ORCID: https://orcid.org/0009-0008-5130-3876)
- Zhichun Feng (ORCID: https://orcid.org/0000-0003-2634-0292)
- Yan Xia (ORCID: https://orcid.org/0000-0002-5323-0614)
- Xiaobing Wu (ORCID: https://orcid.org/0000-0003-0203-2113)
- Xin Li (ORCID: https://orcid.org/0009-0003-2751-1169)
- Wenhui Ma (ORCID: https://orcid.org/0000-0002-6454-8353)
Institutions
- Chinese PLA General Hospital (CN)
- Beijing University of Technology (CN)
- National Center on Birth Defects and Developmental Disabilities (US)
- Annoroad Gene Technology (China) (CN)
- Beijing Center for Disease Prevention and Control (CN)
Publication Details
- Journal
- Molecular Medicine
- Published
- 2025-04-29
- DOI
- https://doi.org/10.1186/s10020-025-01207-4
- Citations
- 2
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 3.25
Funders
- Hebei Medical University
- National Key Research and Development Program of China