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The Children's Hospital Of Philadelphia: New Gene Searching Method Uncovers Possible New Targets For Crohn's Disease Drugs
Friday, 27 February 2009 00:00

Discovering the different genes that contribute to a complex disease is like searching in the proverbial haystack for an unknown number of needles -- some much smaller than others, often blending into the background, and many of them widely separated from each other. But if some needles are linked to each other by fine threads, you might pull out clumps of them together.

Using a novel approach that combines a statistical tool that identifies genes interacting on the same biological pathways with highly automated gene-hunting techniques that scan the whole genome, an international team of researchers has discovered new genes involved in Crohn's disease. Crohn's disease is a chronic and painful condition caused by inflammation of the gastrointestinal tract. The researchers, led by scientists at The Children's Hospital of Philadelphia, say their approach broadens the power of gene discovery studies to ferret out potential targets for disease treatments.

In a complex disorder such as Crohn's disease, many different genes interact to cause the illness. Research over the past few years have identified many of the genes with the strongest effects, but many other genes with important roles may produce weaker or ambiguous signals in the large-scale studies, and go overlooked. "Our pathway-based approach aggregates information from multiple sources to detect modest effects from genes associated with each other," said study leader Hakon Hakonarson, M.D., Ph.D., director of the Center for Applied Genomics at Children's Hospital.

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Stem Cell Treatment For Crohn's Disease
Saturday, 21 February 2009 00:00
Cellular therapy with stem cells is revolutionizing the focus of treatment of many serious diseases. Replacing the cells of damaged tissue with other new cells from the same patient is already a reality. This is the basis of cellular therapy and regenerative medicine, the latest great advance in biomedicine. In this line, Hospital ClĂ­nic, Barcelona is leading the world in the application of an innovative cellular therapy that uses stem cells to treat Crohn's disease, a chronic genetic disease that affects 1% of the population in Spain and which has considerable impact on the quality of life of the patients. The procedure is based on an autologous bone-marrow transplant (when patients receive a transplant of their own stem cells) and now constitutes a treatment option to cure an intestinal disease that sometimes does not successfully respond to drugs and requires highly complex surgery that does not provide a cure.
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Mutation That Causes Inflammatory Bowel Disease Identified By Scientists At Scripps Research
Thursday, 12 February 2009 00:00
A team of scientists at The Scripps Research Institute has linked a mouse mutation to an increased susceptibility for developing inflammatory bowel disease -- represented in humans as Crohn's disease and ulcerative colitis, which together are estimated to affect more than a million people in the United States. The findings may one day lead to new and better treatments for the disease.

The work was published in the February 6, 2009 Early Edition of the Proceedings of the National Academy of Sciences (PNAS).

Humans have a gene that is very similar to the mouse gene, called Mbtps1, and in certain rare instances, mutations of this gene may contribute to IBD in humans. The disease is associated with painful ulcers and bleeding in people's intestines and can place them at greater risk for colon cancer. Although common, the disease is still somewhat mysterious. The Scripps Research study sheds light on a major mechanism through which it may develop.

"We are just beginning to get a sense of the complexity of inflammatory bowel disease as far as humans are concerned," says Bruce Beutler, M.D., who is the chair of the Scripps Research Department of Genetics.

Scientists have known for a long time that IBD is linked to genetics - it runs in families, for instance. However, there seems to be no single gene responsible. More likely, says Beutler, mutations in many different genes have additive effects and cause people to develop variably severe forms of the disease. One of the long-term goals of his laboratory is to identify these genes and the main biological processes they control.
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