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Gene therapy hope for blindness

Press Association logoPress Association 20/03/2017 John von Radowitz

A gene therapy treatment for the most common cause of blindness in young people is being tested in a group of British trial patients.

X-linked retinitis pigmentosa (XLRP) is currently incurable and leads to slow and irreversible vision loss.

On March 16, a 29-year-old man became the first patient to undergo the gene therapy procedure at Oxford Eye Hospital.

The trial team hopes to recruit up to 30 patients for the study, which is designed to assess safety and tolerability rather than effectiveness.

The aim is to use a virus to carry corrective DNA into cells at the back of the eye.

To make the treatment work, scientists have to reprogram a gene called RPGR to make it more stable while not affecting its function.

The instability of RPGR in the retina has previously been a major obstacle to developing gene therapy for the condition.

Trial leader Robert MacLaren, Professor of Ophthalmology at Oxford University, said: "The effect of RPGR-related disease on families with retinitis pigmentosa is devastating and we have spent many years working out how to develop this gene therapy.

"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."

The study is being run by NightstaRx Ltd (Nightstar), a spin-out company developing gene therapies for inherited retinal diseases, and scientists from Oxford University.

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