Nobel Winner’s Stem Cells to Be Tested in Eye Disease Next Year

Posted: October 9, 2012 at 6:13 am

Thomas Perlmann of Karolinska Institute presents Sir John B. Gurdon of Britain and Shinya Yamanaka of Japan as winners of the 2012 Nobel Prize in medicine or physiology. The prize committee at Stockholms Karonlinska institute said the discovery has revolutionized our understanding of how cells and organisms develop. Photograph by Bertil Enevag Ericson/Scanpix/AP Photo

Stem cells derived from a mouses skin won Shinya Yamanaka the Nobel Prize yesterday. 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 blindness, Yamanaka told reporters in San Francisco yesterday.

Companies including Marlborough, Massachusetts-based Advanced Cell Technology Inc. (ACTC) are already testing 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 B. Gurdon, 79, a professor at the University of Cambridge who shared the Nobel with Yamanaka yesterday, said in an interview in London.

Yamanaka and Gurdon shared the 8 million Swedish kronor ($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.

In a study published in 1962, Gurdon took a cell from a tadpoles gut, extracted the nucleus, and inserted it into the egg cell of an adult frog whose own nucleus had been removed. That reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.

Yamanaka, 50, a professor at Kyoto University, built on Gurdons 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 Parkinsons by providing replacement cells.

The implications for regenerative medicine are obvious, R. Sanders Williams, president of the Gladstone Institutes in San Francisco, where Yamanaka is a senior investigator, said in a telephone interview. Skin cells can be converted to any other cell you want -- skin to brain or skin to heart, skin to insulin-producing.

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Nobel Winner’s Stem Cells to Be Tested in Eye Disease Next Year

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