Using a modified virus to treat vision loss from a genetic mutation
Mito-Targeted AAV to treat Leber Hereditary Optic Neuropathy caused by ND4 mutations
This study is exploring a new gene therapy that aims to help people with Leber hereditary optic neuropathy (LHON) by delivering a healthy version of a gene to their cells, which could help improve their vision, and there may be chances for eligible patients to join clinical trials.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project ID | NIH-10554399 on NIH RePORTER |
What this research studies
This research focuses on developing a new gene therapy approach to treat Leber hereditary optic neuropathy (LHON), a condition caused by mutations in mitochondrial DNA. The team has created a modified adeno-associated virus (AAV) that can deliver the normal ND4 gene directly to the mitochondria of affected cells, potentially reversing vision loss. The research will involve testing this therapy in cultured human cells, as well as in animal models, to assess its safety and effectiveness. Patients may have the opportunity to participate in clinical trials if they meet specific criteria related to their genetic condition.
Who could benefit from this research
Good fit: Ideal candidates for participation would be individuals diagnosed with Leber hereditary optic neuropathy due to ND4 mutations.
Not a fit: Patients with LHON caused by mutations other than ND4 may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could restore vision in patients suffering from LHON caused by ND4 mutations.
How similar studies have performed: Previous research has shown promise in using modified viral vectors for gene therapy, indicating potential success for this novel approach.
Where this research is happening
Coral Gables, United States
- University of Miami School of Medicine — Coral Gables, United States (Active)
Researchers
- Principal investigator: Porciatti, Vittorio — University of Miami School of Medicine
- Study coordinator: Porciatti, Vittorio
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.