Development of Two In Vitro <i>ND1</i> -LHON Models for Evaluating Gene Therapy Efficacy
The study demonstrates the utility of transmitochondrial cybrids and iPSC-derived RGCs as reliable in vitro models for studying ND1-related LHON. The rAAV-ND1 gene therapy effectively restored mitochondrial function, highlighting its potential as a treatment for LHON caused by ND1 mutations. These findings underscore the value of in vitro systems for evaluating therapies when robust animal models are unavailable.
Authors
- Yuefeng Liu (ORCID: https://orcid.org/0000-0001-9823-3811)
- Yue Zhang (ORCID: https://orcid.org/0000-0003-3568-0395)
- Jun Jie Yuan (ORCID: https://orcid.org/0000-0002-8265-0239)
- Xin Li (ORCID: https://orcid.org/0000-0002-9466-2659)
- Zhang Chen (ORCID: https://orcid.org/0000-0001-5531-0872)
- Yong Zhang
Institutions
- Hubei University of Medicine (CN)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2025-10-23
- DOI
- https://doi.org/10.1167/iovs.66.13.34
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00