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New stem cell approach for blindness trialled successfully in mice

4 January 2013

Blind mice can see again, after Oxford University researchers transplanted developing cells into their eyes and found they could reform the entire light-sensitive layer of the retina – similar to replacing the film at the back of a camera.

Videos show the nocturnal mice, which once didn’t notice the difference between light and dark at all, now run from the light and prefer to be in the dark – as mice with normal vision.

The researchers believe the approach shows the potential of cell transplantation for restoring vision and may have relevance for treating patients with retinitis pigmentosa, a condition in which the light-sensing cells in the retina gradually die leading to progressive blindness.

The research was led by Professor Robert MacLaren in the Nuffield Department of Clinical Neurosciences at the University of Oxford, together with Dr Mandeep Singh, an eye surgeon from the National University Hospital of Singapore who is currently undertaking PhD studies in Oxford. The findings are published online in the journal PNAS.

The researchers worked with mice that are blind due to complete loss of the light-sensing photoreceptor cells in their retinas, as this is the most relevant mouse model for treating patients who are blind from retinitis pigmentosa.

After two weeks, the researchers showed the stem cells transplanted into the eye had re-formed a full light-detecting layer on the retina and the mice could see.

A pupil constriction test showed that, of the 12 mice that received the cell transplant, 10 showed improved pupil constriction in response to light. This shows that the retinas of the mice were sensing the light once more, and this was being transmitted down the optic nerve to the brain.

The cells used were mouse ‘precursor’ cells that are on an initial path towards developing into retinal cells.

Dr Singh said: ‘We found that if enough cells are transplanted together, they not only become light sensing but they also regenerate the connections required for meaningful vision.’

Professor MacLaren explains: ‘Stem cells have been trialled in patients to replace the pigmented lining of the retina, but this new research shows that the light-sensing layer might also be replaced in a similar way. The light-sensing cells have a highly complex structure and we observed that they can resume function as a layer and restore connections after transplantation into the completely blind retina.’

In looking forward towards potential cell treatments for blindness in humans, Professor MacLaren explains that they would like to use induced pluripotent stem cells or iPS cells. These are stem cells that have been generated from the patient’s own cells, such as skin or blood cells, and can then be directed to form precursors of the retina cells.

Professor MacLaren says that this has been achieved by others: ‘All the steps are there for doing this in patients in the future.’ The next step is to find a reliable source of cells in patients that can provide the stem cells for use in such transplants, he says.

While these are more long-term developments to work towards, Professor MacLaren says ‘Our study shows what we could achieve with a cell-based approach.’

‘We have shown the transplanted cells survive, they become light-sensitive, and they connect and reform the wiring to the rest of the retina to restore vision,’ he says. ‘The ability to reconstruct the entire light sensitive layer of the retina using cell transplantation is the ultimate goal of the stem cell treatments for blindness we are all working towards.’

For more information please contact Professor Robert MacLaren on  robert.maclaren@eye.ox.ac.uk

Or the University of Oxford press office on +44 (0)1865 280530 or press.office@admin.ox.ac.uk

For a copy of the paper, video or photos, please contact the University of Oxford press office at press.office@admin.ox.ac.uk.

Notes for Editors:

  • The paper ‘Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation’ by Mandeep Singh and colleagues is published online in the journal PNAS.
  • The research is funded by the Oxford Stem Cell Institute at the Oxford Martin School, University of Oxford; the Lanvern Foundation; the Health Foundation; the Royal College of Surgeons of Edinburgh; Fight for Sight; the Special Trustees of Moorfields Eye Hospital and the Singapore National Medical Research Council. Professor MacLaren also receives funding for stem cell research projects from the Medical Research Council and the Wellcome Trust.
  • Oxford University’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.