procedure designed to treat hereditary liver disease, may herald a new era of therapy for genetic diseases
scientists took skin cells from a patient with liver disease and has become a liver cell replacement on a tour of the biological force that promises to transform the way the disease is treated.
The procedure will have to undergo years of testing before it can be used in humans, but if approved, could launch a new era of personalized therapies for genetic diseases.
In Britain, 30,000 people carry a genetic abnormality that causes antitrypsin, a disease that can not be cured by a liver transplant. The operation requires an appropriate organization and costs about £ 500 000, with drugs to prevent rejection by the immune system of the addition of more than £ 20,000 per year for medical expenses.
treat patient's own cells eliminates the need for anti-rejection drugs, reduce the burden of registry services tense and is likely to be cheaper, scientists believe that behind the technique.
"The disease affects very young, even babies, and not always suitable donors for many of these people," said Allan Bradley, former director of the Wellcome Trust Sanger Institute in Cambridge. "These are the first steps, but if this technology can have on the treatment, which will offer great benefits for patients."
The genetic flaw that causes the disease causes the liver cells produce defective versions of a protein. The normal protein circulates in the blood and protects the body's tissues and organs of the routine damage, but in people with antitrypsin deficiency, misfolded proteins accumulate in the liver.
Over time, the disease causes a cirrhotic liver disease and let the other organs vulnerable to injury. Most at risk are the lungs, and many patients develop progressive emphysema as a secondary condition.
In the final step, the scientists used chemicals to make iPS cells in healthy liver cells. When these cells were injected into mice, they met in the liver, where it produces healthy antitrypsin protein and other chemicals released by cells of normal liver.
scientists now hope to work with a major pharmaceutical company and working for human trials. Instead of injecting cells directly into patients, cells is probably encapsulated in a porous bag. This will ensure that patients are not at risk if some turn out to be defective cells and develop into tumor cells.
other scientists are expected to develop the procedure for the treatment of other genetic diseases, including those requiring the correction of mutations at a time.
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