UCL Institute of Ophthalmology
- 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 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...
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 […]Read more...
Current gene and cell therapy clinical trials for sight loss
We are translating our research on inherited and acquired forms of sight loss into clinical trials. Find out about how you can support our work and help develop effective therapies.
We are currently conducting two ground-breaking clinical trials:
Phase I/II trial to assess safety and efficacy of viral RPE65 gene therapy for Leber congenital amaurosis
We began the world's first gene therapy trial in the eye in 2007.
The trial is to test the safety and efficacy of gene therapy using an adeno-associated virus (AAV) to deliver the RPE65 gene to retinal cells in patients who have Leber congenital amaurosis (LCA).
Further patients have been treated, and the latest arm of the study includes younger patients.
The encouraging results from this trial suggest that gene therapy can be an effective treatment for inherited retinal degeneration, and extensive evidence from laboratory research shows that other diseases that lead to blindness may also be treatable using gene therapy.
Each of these therapies requires the development of a new gene therapy vector and independent safety testing
Visit the EyeTherapy information hub for more details on:
Pipeline of gene therapy treatments for sight loss
The results of this trial indicate that gene therapy in the eye is safe and can be effective at treating sight loss.
We are developing a pipeline of gene therapy strategies for a range of other conditions that cause sight loss.
Phase I trial to determine the safety of transplanting stem cell-derived retinal cells
The cells being injected are retinal pigment epithelium (RPE) cells that were grown from human embryonic stem cells in a dish, by the American company Advanced Cell Technology Inc.
This trial involves patients who are already severely visually impaired and is designed to show whether the injection of these cells is safe - future studies will be required to determine if cell transplantation can restore sight.
- Cell transplantation and stem cell therapy for sight loss
- Visit the EyeTherapy information hub for more details on the clinical trial of stem cell-derived retinal cells in Stargardt disease and how we are developing cell transplantation for sight loss.
- The Guardian: Stem cell scientists take hope from first human trials but see long road ahead
Page last modified on 30 nov 12 10:50