Stem Cells Restore Sight to Mice

Researchers using a virus to deliver corrective genes to malfunctioning cone cells have cured a common form of blindness in mice. Excitingly, the mice retained their newfound sight -- which tests suggested was fully normal -- for months after the treatment.

The discovery shows that it's possible to target and rescue cone cells, the most important cells for visual sharpness and color vision in people. [...]

The ability to deliver a gene specifically to cone cells has implications for all blinding diseases, not just inherited ones,
Hauswirth said. "Even in two very common types of blindness, age-related macular degeneration and diabetic retinopathy, if you can target cones you might be able to rescue that vision," he said.

As always, it's a long ways from mice to people, but some research does make that journey, and the genetic defect underlying achromotopsia -- the type of blindness studied -- is the same in humans and mice.

The therapy was made possible in part by the discovery several years ago at the Jackson Labs of mice with achromatopsia. I spoke to a Jackson lab official on the way to the Biotechnology
Industry Conference, and with several lab scientists yesterday, and they talked about a boom over the last few years in mice bred for specific genetic conditions. Granted, it's their job to be excited about that, but it sounds like a great many studies like this will soon be possible.

Related Wired coverage here and here.

Maine mice play role in eye disease cure [Boston Globe]
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