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Gene therapy success in monkeys may help cure human blindness one day


June 24, 2011 - London

Researchers have successfully used gene therapy to help blind monkeys regain their sight, a finding that could someday be applied to humans.

Gene therapy has only been used to partially restore sight in people who are blind from defects in the pigmented layer of the eye, providing the same treatment for those who are blind through loss of photoreceptor cells - the cells that respond to light - has proven a greater challenge.

Luk Vandenberghe at the University of Pennsylvania in Philadelphia, and colleagues tried to find out whether it would be possible to deliver genes that would eventually restore photoreceptor cells, reports New Scientist.

They used two adeno-associated viruses, called AAV2 and AAV8, to deliver genes into monkey retinas.

The viruses were marked with a green fluorescent Protein. By monitoring the green markers, the researchers were able to assess the minimum dose at which the viruses could deliver working genes into the retinal cells.

A dose of AAV8 performed best, presenting an important step in using gene therapy to treat and restore photoreceptor cells in humans.

The study has been published in Science Translational Medicine.

ANI

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