Gene and Cell Therapy Group
- Research team
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- 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
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 RPE65 Gene Therapy Trial Shows Benefit in People with Leber Congenital Amaurosis Type 2 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 for Retinitis Pigmentosa!
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...
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 for people with Stargardt Macular Degeneration
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