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Bloomberg.com: News: "Harvard Team Makes 10 Disease-Bearing Stem Cell Lines (Update2)

By Rob Waters

Harvard Team Makes 10 Disease-Bearing Stem Cell Lines (Update2)

By Rob Waters

Aug. 7 (Bloomberg) -- Harvard University scientists have made lines of stem cells, able to turn into any other cell in the body, from bits of skin or blood of 10 patients with genetic diseases including muscular dystrophy and juvenile diabetes.

The findings will help researchers decipher the workings of these diseases, enabling them to study what happens as cells that carry a condition's genetic seeds develop and age. The lines will be made available for a ``nominal fee'' to researchers around the world, the Harvard scientists said.

Teams at the Harvard Stem Cell Institute in Cambridge, Massachusetts, created the lines using a technique that reprograms cells to give them the same power as those from embryos to become any of the roughly 210 cell types in the body. Their advance was described in a paper appearing today in the journal Cell.

The advance will ``allow researchers for the first time to get access'' to cells that are defective in a particular disease ``and to watch the disease progress in a dish, to watch what goes right or wrong,'' said Doug Melton, a Harvard cell biologist and co-director of the institute.

The Harvard teams created the new lines from tissue taken from 10 patients who ranged in age from a 3-month-old child with a form of immune deficiency sometimes known as ``bubble boy disease'' to a 57-year-old with Parkinson's. Last week, another Harvard team said they'd performed the same feat using the skin of two patients in their 80s with the neurodegenerative condition known as Lou Gehrig's disease.

Yamanaka Technique

The technique used to create the stem cells, developed by Shinya Yamanaka of Kyoto University in Japan, has captivated scientists and transformed the research they're performing. The method involves using viruses to insert four different genes into skin cells. The genes turn on a process that causes the cells to revert to a primordial state similar to embryonic stem cells.

Yamanaka announced his breakthrough two years ago at a scientific meeting in Toronto, when he described how he had been able to endow skin cells from mice with the power of those from embryos. Other advances followed rapidly. Last November, two research teams, one led by Yamanaka and the other by James Thompson of the University of Wisconsin in Madison, announced independently that they'd done the same thing with the skin of living people.

Research teams around the world have rushed to use Yamanaka's technique for creating what he calls induced pluripotent stem cells, or IPS cells, for two key reasons. It is relatively easy and inexpensive to perform and it doesn't require the use of human embryos or unfertilized eggs, both of which can be difficult to obtain.

Ethical Concerns

Because human embryos aren't used or harmed to create the IPS cells, the method sidesteps ethical concerns that have dogged researchers. Religious and political leaders including President George W. Bush have objected to traditional stem cell research because embryos are destroyed in the process of creating the lines.

Still, because Yamanaka's technique uses viruses and genes that are known to cause cancer, lines created with this method can't be used as treatments. They will allow researchers to peer into the complex molecular and genetic processes that occur in defective cells as they develop, giving them a greater understanding of how and why disease begins.

``We are so ignorant at the moment we don't even know if when patients gets diabetes, they all get it the same way,'' Melton said in a conference call yesterday with reporters. ``There could be 50 different ways of getting Type 1 diabetes.''

Next Steps

George Daley, the lead author of today's paper and a researcher at Children's Hospital in Boston who studies blood diseases, said he and his colleagues will now take the newly minted stem cells and coax them to become blood cells of various types.

He said he hopes that by comparing them with normal healthy blood, ``we can find the particular development points where the defects arise and we can look at gene-repair strategies.''

He and other scientists also will be able to test thousands of existing drugs to see whether any of them remedy the defects, he said.

Daley said the new lines, and those developed in the future, will be maintained in a new laboratory at Massachusetts General Hospital in Boston. Setting up the lab will enable other researchers to obtain the Harvard cells for their own experiments, something that didn't happen quickly after embryonic stem cells were first isolated in 1998, he said.

To contact the reporter on this story: Rob Waters in San Francisco at rwaters5@bloomberg.net.
Last Updated: August 7, 2008 15:03 EDT