1st patient injected with gene therapy for X-linked retiniti
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Researchers have injected their first patient with a virus engineered to remodel the gene responsible for X-linked retinitis pigmentosa (XLRP).

“If successful, this gene therapy has the potential to transform the lives of many patients,” said David Fellows, chief executive officer, Nightstar, a gene therapy company in Oxford, according to an Oxford University press release.

The injection took place as part of a multicenter open-label study designed to enrolat least 24 male patients in a 12-month trial of safety and tolerability. It is the first in the world to test a treatment for retinitis pigmentosa caused by the retinitis pigmentosa GTPase regulator (RPGR) gene, the press release said.

One of the leading causes of blindness in young people, retinitis pigmentosa is currently untreatable and leads to a slow and irreversible loss of vision due to the loss of rods and cones.

The most prevalent form of XLRP is caused by mutations in the RPGR gene, accounting for over 70% of XLRP patients....

http://ophthalmologytimes.modernmedicine.com/ophthalmologytimes/news/gene-therapy-trial-launched-x-linked-retinitis-pigmentosa?page=0,0
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