Targeted AAV6 gene therapy restores corneal endothelial function in three hereditary corneal dystrophies - PubMed
5 hours ago
- #gene therapy
- #corneal dystrophy
- #AAV6
- Targeted AAV6 gene therapy shows promise in treating three hereditary corneal dystrophies: MCD, FECD, and CHED.
- A refined intracameral injection method enables effective endothelial transduction without corneal puncture.
- AAV6 therapy supports sustained transgene expression for over 18 months with no adverse immune responses.
- In MCD mice, AAV6-Chst5 reduces corneal opacification and restores keratan sulfate levels.
- In FECD mice, AAV6-Col8a2 prevents corneal opacity in 87.5% of treated eyes.
- In CHED models, AAV6-Slc4a11 resolves corneal edema within 7 days.
- Single-cell RNA sequencing identifies Wnt5a as a key factor in MCD pathogenesis.