6 · Emerging therapies — cellular, gene & innovative
Corneal gene therapy
Review — modulators of corneal healing › 6 · Emerging therapies — cellular, gene & innovative › Corneal gene therapy
Corneal gene therapy
Viral (AAV, lentivirus) / non-viral vectors; gene augmentation, RNAi/antisense, CRISPR editing
Oxford V (preclinical)
Investigational
Future · frontier
Mechanism of actionThe avascular, immune-privileged and accessible cornea = an ideal target. Strategies: anti-fibrotic/anti-angiogenic genes (decorin, sFlt-1, PEDF) ↓scarring/neovascularisation; correction of dystrophies (TGFBI allele-specific silencing, endothelial augmentation); CRISPR editing of dominant mutations. Strong endothelial tropism of AAV6.
IndicationsFibrotic scarring after injury/surgery, neovascularisation, hereditary dystrophies (TGFBI, CHED/Fuchs), graft survival.
Route & dosageInvestigational — intrastromal injection, topical, anterior chamber, or ex vivo graft transduction. No human dosing established.
Level of evidenceOxford V (preclinical / ex vivo human corneas) · Scale: Investigational. No approved corneal product (contrast: retinal Luxturna).
| FDA / EU MA | None — worldwide research only. |
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Key studies: AAV6 gene therapy restoring endothelial function (Cell Rep Med 2026) — PMID 41850243 ; ex vivo AAV transduction of the human limbus; TGFBI editing programmes.