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Gene therapy used to cure blindness in monkeys

27 June 2011
Appeared in BioNews 613

A group of US scientists has successfully used a virus to deliver genes into monkey retinas that could potentially restore damaged photoreceptor cells. The findings could lead to gene therapies being developed to treat blindness and other eye conditions in humans.

In previous trials, researchers have used the AAV2 serotype of the adeno-associated virus to partially cure congenital blindness in retinal pigment epithelial cells. Treating problems in the photoreceptor layer of retinal cells, which are faulty in most inherited forms of blindness, has however proven far more difficult.

Dr Luk Vandenberghe and his colleagues at the University of Pennsylvania, Philadelphia, used both the AAV2 and AAV8 forms of the virus to deliver the foreign genes into monkeys and used a green fluorescent protein to test what dose of virus was required. They measured the success of delivery by detecting the fluorescent protein in the eyes of treated animals. AAV8 in this case worked slightly better than AAV2 serotype.

The eye is one of the few parts of the body where the insertion of foreign genes does not trigger inflammatory immune responses, making it a suitable target for gene therapies.

The research was published in the journal Science Translational Medicine.

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