A A A

EyeTherapy Blog News

Registration for Retina Day 2015 now open!

Wed, 10 Jun 2015 11:37:25 +0000

It’s that time once agin for our annual research day for patients and the public. Retina Day 2015 is a free, one day event is organised by the Gene and Cell Therapy Group, UCL Institute of Ophthalmology and NIHR Moorfields Biomedical Research Centre. Come along to: * Hear about some of the latest innovations in research […]

Read more...

UCL Gene Therapy Trial shows Benefit for up to Three Years After Treatment

Tue, 05 May 2015 14:44:39 +0000

We are delighted to be able to announce that yesterday, Monday 4th May, the long-term results of our RPE65 gene therapy trial for Leber Congenital Amaurosis Type 2 (LCA2) were published in the prestigious New England Journal of Medicine. Begun in 2007, this was the world’s first-in-human trial of gene therapy to treat an inherited […]

Read more...

UCL researchers solve a major riddle of retinal degeneration research!

Mon, 26 Jan 2015 10:11:36 +0000

Today a paper published in Nature Communications from the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology has shed light on why, until now, it has not been possible to effectively restore vision in rd1 mice – the world’s major model for retinitis pigmentosa (RP). The rd1 mouse is a model of […]

Read more...

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