Gene therapy for corneal disease

Damage to the cornea can cause sight loss that only transplants can reverse - but these transplants often fail. Find out how you can support our work and help develop effective therapies.

The cornea, the transparent window at the front of the eye, can become damaged as a result of genetic conditions such as Fuchs endothelial dystrophy, complex conditions like keratoconous, and injury. In some cases this damage requires a corneal transplant to repair, which are subject to both immune rejection and graft failure. We are developing ways of improving the quality of corneas used for transplant, and reducing the chances of immune rejection, using gene therapy.

Improving the quality of donor corneas for transplantation


Our aim is to take human corneas that have been donated but are of unsuitable quality for organ transplantation, infect them temporarily with a viral vector carrying a gene that improves the cornea's health, then proceed with the transplant as usual.

We have previously shown that both vectors based on both adenovirus (McAlister et al IOVS 2005) and lentivirus (Bainbridge et al Gene Therapy 2001) can deliver genes to the corneal endothelial cells.

We are currently focussing on delivering genes to human corneal cells in culture using lentiviral and AAV-based vectors, with the aim to improve corneal transplant quality.

Lentiviral vectors can efficiently deliver genes to cells in the cornea
Lentiviral vectors can efficiently deliver genes to cells in the cornea - four images showing a reporter gene, encoding green fluorescent protein, can be efficiently delivered to cells in the cornea

Preventing immune rejection of corneal transplants

A common cause of cornea transplant failure is rejection of the graft cornea by the host immune system

We are developing strategies to help reduce the likelihood of immune rejection, seeking to deliver genes that would regulate the host immune response to the transplant.

Page last modified on 07 nov 12 17:04

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