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Gene therapy for blindness clears hurdle in mice

ount of protein formed by mutated rhodopsin, that may be sufficient to maintain vision in people who are affected by retinitis pigmentosa," Gorbatyuk said. "The second step, introducing the normal gene to the retina, will show whether we are able to restore vision in this model or not."

If both steps are perfected, scientists plan to study the treatment in a larger animal model and then possibly move to a human clinical trial.
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Source:University of Florida


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