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Gene therapy restores vision in blind mice

09 October 2017

By Dr Kimberley Bryon-Dodd

Appeared in BioNews 921

Gene therapy has restored some vision in mice blinded by retinitis pigmentosa, an inherited degenerative eye disease.

Retinitis pigmentosa is the most common cause of blindness in young people, and no effective treatments currently exist for patients with little sight left.

'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,' said Dr Samantha de Silva at the University of Oxford, the lead author of the study.

Diseases like retinitis pigmentosa cause gradual loss of light-sensitive cells in the eye's retina, eventually leading to blindness. Rather than trying to restore these cells, researchers used a viral vector to introduce a gene for melanopsin, a light-sensitive protein naturally found in the eye, into the remaining retinal cells.

The cells then produced melanopsin and became able to sense and respond to light, and send visual signals to the brain. The changes lasted longer than a year, around the lifetime of the mice used in the study.

'Our next step will be to start a clinical trial to assess this in patients,' said Dr de Silva.

The authors are hopeful that this common form of blindness may be able to at least be partially restored in the future by using this gene therapy technique.

'The effect of retinitis pigmentosa on families with the disease is devastating and we have spent many years working out new ways to slow the loss of sight and to begin restoring it,' said Professor Robert MacLaren also at the University of Oxford, whose group led the research. 'This new approach is exciting because by using a human protein that is already present in the eye we reduce the chances of causing an immune response.'

Professor Alan Boyd, President of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians, who was not involved in the study, said that no satisfactory treatments currently exist for patients with end-stage retinal degeneration, who have little sight left. Although human clinical trials could be a minimum of two to three years away, he said: 'These results are very encouraging and could lead to a potential treatment for end-stage blindness in humans.'

The study was published in the journal PNAS.


16 October 2017 - by Rikita Patel 
The US Food Drug and Administration advisory committee has backed the use of gene therapy to treat a hereditary disease for the first time...

22 May 2017 - by Dr Kimberley Bryon-Dodd 
A small, preliminary clinical trial has demonstrated safe and well-tolerated gene therapy to treat wet age-related macular degeneration...
03 May 2016 - by Sarah Gregory 
A trial of gene therapy for choroideremia, a rare form of inherited blindness, has partially restored the vision of several patients...
01 February 2016 - by Kulraj Singh Bhangra 
Researchers have used the CRISPR/Cas9 genome-editing technique to correct a genetic mutation that causes blindness...
24 August 2015 - by Antony Blackburn-Starza 
An experimental form of gene therapy to treat blindness has been showed to restore some vision in mice....
15 December 2014 - by Dr Rosie Gilchrist 
Scientists have used gene therapy to restore some sight to animals with visual impairment similar to retinosa pigmentosa, a type of human blindness...

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