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News
Photoreceptor transplants restore vision in mice
Key achievements
The world's first eye gene therapy trial - JWB Bainbridge et al. N Engl J Med. 2008
Cell therapy repairs the retina - Pearson RA et al Nature 2012
The first proof-of-concept in eye gene therapy - RR Ali et al. Nat. Genet. 2000
Our latest blog posts
It’s OK to ask about clinical research – an NIHR campaign
‘Defining future eye research’ – a chance for you to help tackle slight loss
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).
Initial results published in 2008 (Bainbridge et al. NEJM 2011) suggest that the technique is safe and can restore night-vision in some cases.
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
We are conducting Europe's first clinical trial looking at the safety of injecting retinal cells, derived from embryonic stem cells, into the eyes of patients with Stargardt macular dystrophy.
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
