Skin transformed into liver cells to treat an inherited disease

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.

an article published in the journal Nature, the team led by researchers at the Sanger Institute, describe how they tackled the science of stem cells is advancing rapidly to find a new way of do against the disease. They embarked on a process that took months, from beginning to end, and consisted of several steps based on the recently developed genetic techniques.

• In the first stage of the procedure, the team took skin cells from a patient with antitrypsin deficiency responsible for virus proteins are used to reprogram the most versatile cells, called pluripotent stem cells induced (iPS). They are very similar to embryonic stem cells can develop in almost any tissue of the body.

Researchers then began to fix the genetic defect in the cells, the use of enzymes to the house on the defective gene, cut and replace it with a strand of DNA correctly. This feat requires exquisite precision to ensure that only one of the three billion pairs of letters that make up the human genetic code has changed. Other changes in the patient's DNA could lead to serious medical problems.

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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