Nobel winner's stem-cell technology to be tested on macular degeneration

— Stem cells derived from a mouse’s skin won Shinya Yamanaka the Nobel Prize on Monday. Now researchers in Japan are seeking to use his pioneering technology for an even greater prize: restoring sight.

Scientists at the Riken Center for Developmental Biology in Kobe plan to use so-called induced pluripotent stem cells in a trial among patients with macular degeneration, a disease in which the retina becomes damaged, resulting in loss of vision, Yamanaka told reporters in San Francisco on Monday.

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http://www.arkansas…"> 2 stem-cell research pioneers win Nobel

Companies including Pfizer are already planning trials of stem cells derived from human embryos. The Japanese study will be the first to use a technology that mimics the power of embryonic cells while avoiding the ethical controversy that accompanies them.

“The work in that area looks very encouraging,” John Gurdon, 79, a professor at the University of Cambridge who shared the Nobel with Yamanaka, said in London.

Yamanaka and Gurdon shared the $1.2 million award for experiments 50 years apart that showed that mature cells retain in latent form all the DNA they had as immature stem cells, and that they can be returned to that potent state, offering the potential for a new generation of therapies against hard-to-treat diseases such as macular degeneration.

Yamanaka, 50, a professor at Kyoto University, built on Gurdon’s work by adding four genes to a mouse skin cell, returning it to its immature state as a stem cell with the potential to become any cell in the body. He dubbed them induced pluripotent stem cells.

The technology may lead to new treatments against diseases such as Parkinson’s by providing replacement cells.

Scientists first must ensure that the cells are safe, Yamanaka said in a video appearance from Japan on Monday at the San Francisco news conference organized by Gladstone. One concern is that stem cells could grow out of control, leading to cancers.

“We need to double check we don’t see any severe side effects in patients after transfer,” he said. “That’s where we have been spending most of our time. We are getting closer and closer. In some diseases, like macular degeneration, it’s almost ready to go.”

Takahashi and colleagues have already succeeded in transplanting retinal cells into mice. None of the animals developed cancer, the report said.

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