Preclinical Assessment of Mitochondrial-Targeted <i>ND4</i> Gene Therapy for Leber Hereditary Optic Neuropathy
Intravitreal delivery of MTSAAV/hND4 in rodents resulted in favorable biodistribution, with vector DNA largely confined to the injected eye and minimal systemic exposure. No vector-related adverse effects were observed based on functional, anatomic, and histopathological assessments, supporting the ocular safety of this gene therapy strategy. These findings provide preclinical evidence supporting the advancement of MTSAAV-mediated ND4 gene therapy toward clinical trials for LHON.
Authors
- Hong Yu (ORCID: https://orcid.org/0000-0002-1272-4511)
- Byron L. Lam (ORCID: https://orcid.org/0000-0002-1233-2366)
- Annie Chang (ORCID: https://orcid.org/0000-0003-4692-6338)
- Yuan Liu (ORCID: https://orcid.org/0000-0002-3421-3579)
- Zixuan Hao
- Xuemin Jin (ORCID: https://orcid.org/0000-0002-1850-6996)
- Yuan Dong (ORCID: https://orcid.org/0000-0003-4336-2813)
- Vittorio Porciatti (ORCID: https://orcid.org/0000-0002-1592-3532)
Institutions
- University of Miami (US)
- First Affiliated Hospital of Zhengzhou University (CN)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2025-11-24
- DOI
- https://doi.org/10.1167/iovs.66.14.58
- Citations
- 1
- Primary Topic
- Mitochondrial Function and Pathology
- Type
- article
- Field-Weighted Citation Impact
- 0.81
Funders
- University of Miami