Oxford News Release - Gene therapy shows promise for reversing blindness

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In a laboratory study in Oxford, researchers have shown how it might be possible to reverse blindness using gene therapy to reprogram cells at the back of the eye to become light sensitive. Most causes of untreatable blindness occur due to loss of the millions of light sensitive photoreceptor cells that line the retina, similar to the pixels in a digital camera.

The remaining retinal nerve cells which are not light sensitive however remain in the eye. Samantha de Silva and colleagues used a viral vector to express a light sensitive protein, melanopsin, in the residual retinal cells in mice which were blind from retinitis pigmentosa, the most common cause of blindness in young people.

The mice were monitored for over a year and they maintained vision during this time, being able to recognise objects in their environment which indicated a high level of visual perception. The cells expressing melanopsin were able to respond to light and send visual signals to the brain. The Oxford team has also been trialling an electronic retina successfully in blind patients, but the genetic approach may have advantages in being simpler to administer.

The research was led by Professors Robert MacLaren and Mark Hankins at the Nuffield Laboratory of Ophthalmology in Oxford. Samantha de Silva, the lead author of the study said: ‘There are many blind patients in our clinics and the ability to give them some sight back with a relatively simple genetic procedure is very exciting. Our next step will be to start a clinical trial to assess this in patients.’
The full paper, 'Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy', can be read in PNAS.

For further information, please contact Chris McIntyre in the University of Oxford press office at christopher.mcintyre@admin.ox.ac.uk or on+44 (0)1865 270 046.

Notes to editors:

The Nuffield Department of Clinical Neurosciences has an established research and teaching portfolio with a national and international reputation for excellence. It comprises five sections: the Nuffield Division of Anaesthetics, the Division of Clinical Neurology, The Centre for Functional Magnetic Imaging of the Brain, the Nuffield Laboratory of Ophthalmology and the Centre for the Prevention of Stroke & Dementia. The Department is based in the John Radcliffe Hospital and has developed a highly integrated and interdisciplinary environment in which research, teaching, clinical training and clinical care interact. This enables new approaches to the understanding, diagnosis and treatment of brain diseases. 

Oxford University Hospitals NHS Foundation Trust (OUH) is one of the largest acute teaching trusts in the UK, with a national and international reputation for the excellence of its services and its role in patient care, teaching and research. The Trust supports world-leading research programmes in cardiovascular diseases, musculoskeletal disorders, neurological disorders such as Parkinson’s and Alzheimer’s through its designation as one of the UK’s five comprehensive biomedical centres and units. It works in close partnership with the University of Oxford and is a leading centre for cancer, neurosciences, diabetes, genetics and many other fields. Research themes of particular strength are: cancer, cardiovascular science, diabetes, endocrinology & metabolism, infection and immunology, musculoskeletal science, neuroscience and reproduction and development. As of October 1 2015, the Trust was awarded Foundation Trust status. This decision comes after the Care Quality Commission gave OUH an overall rating of 'Good' in May 2014, and after scrutiny of the Trust's quality, finances, service delivery and governance arrangements by the NHS Trust Development Authority and Monitor. The Trust has been designated as a major trauma centre and is one of four UK centres for craniofacial surgery and The Trust employs over 12,000 staff and consists of four hospitals: the Churchill Hospital, John Radcliffe Hospital and Nuffield Orthopaedic Centre in Oxford and the Horton General Hospital in Banbury. www.ouh.nhs.uk 

The University of Oxford’s Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. The University is rated the best in the world for medicine, and it is home to the UK’s top-ranked medical school. From the genetic and molecular basis of disease to the latest advances in neuroscience, Oxford is at the forefront of medical research. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. A great strength of Oxford medicine is its long-standing network of clinical research units in Asia and Africa, enabling world-leading research on the most pressing global health challenges such as malaria, TB, HIV/AIDS and flu. Oxford is also renowned for its large-scale studies which examine the role of factors such as smoking, alcohol and diet on cancer, heart disease and other conditions.

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