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Eye Therapy News

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 […]

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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 […]

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2 Lazy 2 Run? We’re biking it for blood cancer!

Fri, 29 Aug 2014 09:30:05 +0000

  On Sunday 31 August a group of not so elite athletes from the Gene and Cell Therapy group will be taking part in the London Bikeathon 2014 to raise funds for Leukaemia & Lymphoma Research. The 2 Lazy 2 Run CC will be cycling 52 miles – that’s more than a marathon, no mean feet […]

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The Art of Eyes

Thu, 07 Aug 2014 14:23:19 +0000

The eye is an object of great beauty as shown by the Ophthalmologist in their July/August 2014 issue. This month’s issue features a photo essay called The Art of the Eyes and includes examples of the work from a number research labs capturing the complex and beautiful detail of the eye and its cells. The essay includes images […]

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In memoriam

Tue, 05 Aug 2014 16:02:34 +0000

Dr Yoshiki Sasai (1962 – 2014) It is with great sadness today that we remember and pay tribute to our collaborator Dr Yoshiki Sasai. Yoshiki was a world leading stem cell researcher and Deputy Director of the Riken Center for Developmental Biology in Kobe, Japan. Through his hard work and dedication over many years, Yoshiki […]

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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.

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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 at restoring vision in small and large animal models of inherited retinal degeneration.

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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.

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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.

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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.

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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.

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Page last modified on 11 sep 12 14:23