Groundbreaking Gene Therapy for Retinitis Pigmentosa

Retinitis Pigmentosa (RP) is an x-linked, inherited disease that causes the progressive degeneration of rod photoreceptors in the retina. This disease affects ~15,000 people in Britain and slowly leads to an irreversible form of blindness.

There are several genes which can cause RP when mutated. One which is particularly prone to mutations, known as the Retinitis Pigmentosa GTPase Regulator (RPGR), is responsible for maintaining the photoreceptors. In some patients suffering from RP, this gene lacks over half of its DNA code and comprises of only the nucleotides G and A in areas.

A groundbreaking technique has been developed in which a harmless virus is used to deliver a healthy version of the gene containing the correct DNA into the eye. This gene therapy has now been performed for the first time on a blind male suffering from RP. It will, however, be a couple of years before the success of this technique can be certain.

“Changing the genetic code is always undertaken with great caution, but the new sequence we are using has proven to be highly effective in our laboratory studies.” Robert MacLaren, Professor of Ophthalmology at the University of Oxford.

For more information, visit the University of Oxford webpage:

http://www.ox.ac.uk/news/2017-03-17-new-trial-blindness-rewrites-genetic-code

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