Gene and Cell Therapy Group
- Our research programme
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- UCL IoO
- NIHR Moorfields BRC
- Moorfields Hospital
Our research programme
Clinical trials of gene and cell therapy for sight loss
We are currently conducting two ground-breaking clinical trials:
The world's first gene therapy trial in the eye, testing the safety and efficacy of delivering the RPE65 gene to the eyes of patients with Leber congenital amaurosis.
Europe's first trial testing the safety of transplanting stem cell-derived retinal cells into the eyes of patients with Stargardt disease.
A pipeline of gene and cell therapies for sight loss
Gene and cell therapies for inherited sight loss
We are developing a pipeline of potential therapies for which we have
established proof-of-concept, showing that gene therapy and the
transplantation of retinal cells derived from stem cells are effective
restoring vision in small and large animal models of inherited retinal
Gene and cell therapies for age-related macular degeneration (AMD)
We are investigating novel treatments for AMD that build on our understanding of the causes of sight loss in this complex disease. Our programme of laboratory research into AMD covers the mechanisms of sight loss and the development of both gene and cell therapy.
Gene therapy for diabetic eye disease
Diabetic eye disease, including diabetic retinopathy and maculopathy, is the leading cause of vision loss amongst those with diabetes.
We have used several well-established animal models of disease to further our understanding of factors that contribute to vision loss in these diseases, with a view to identifying drug targets and ways to intervene using gene and cell therapy.
Gene therapy for uveitis
We have established proof-of-concept that the delivery of genes encoding anti-inflammatory proteins can reduce damage to the retina in models of uveitis, and are developing new and more effective ways of treating this common cause of sight loss.
Gene therapy for corneal disease
We are developing ways of improving the quality of corneas used for transplant, and reducing the chances of immune rejection, using gene therapy.
Page last modified on 11 sep 12 14:23