- Current clinical trials of gene and cell therapy for sight loss
- Gene and cell therapies for inherited sight loss
- Gene and cell therapies for age-related macular degeneration (AMD)
- Gene therapy for diabetic eye disease
- Gene therapy for uveitis
- Gene therapy for corneal disease
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EyeTherapy Blog News
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...
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 […]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.
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
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 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.
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