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EyeTherapy Blog News

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 […]

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Professor Ali honoured for his contribution to research into retinal disease

Mon, 08 Sep 2014 12:27:36 +0000

              Professor Robin Ali, PhD, Professor of Human Molecular Genetics and Head of the Department of Genetics, UCL Institute of Opthalmology has been awarded the Pioneer Award for his work in proof-of-concept studies that have demonstrated the feasibility of using gene therapy and cell transplantation to treat dysfunction and degeneration of the cells […]

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Achromatopsia might not be as progressive as previously thought

Mon, 08 Sep 2014 11:17:16 +0000

A recent publication from the UCL Institute of Ophthalmology, Moorfields Eye Hospital, and the Medical College of Wisconsin indicates that for the majority of people with achromatopsia, the condition may not be as progressive as previously suggested. Data from this study by Aboshiha et al. demonstrated that for the majority of people with achromatopsia (a […]

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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