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Health, Wellness, Sex and Body Discuss Hemophilia & other blood related disorders/diseases at the General Discussion; Hemophilia B Gene Therapy Breakthrough... In a Breakthrough for Gene Therapy, an Injection Works on Hemophilia B Saturday 10th December, ...

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Old 12-10-2011, 07:02 PM
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Lightbulb Hemophilia & other blood related disorders/diseases

Hemophilia B Gene Therapy Breakthrough...

In a Breakthrough for Gene Therapy, an Injection Works on Hemophilia B
Saturday 10th December, 2011 - A virus carrying a replacement gene for blood clotting was used by University College London researchers to help six patients.
Medical researchers in Britain have successfully treated six patients suffering from the blood-clotting disease known as hemophilia B by injecting them with the correct form of a defective gene, a landmark achievement in the troubled field of gene therapy. Hemophilia B, which was carried by Queen Victoria and affected most of the royal houses of Europe, is the first well-known disease to appear treatable by gene therapy, a technique with a 20-year record of almost unbroken failure. “I think this is a terrific advance for the field,” said Dr. Ronald G. Crystal, a gene therapist at Weill Cornell Medical College. “After all the hype in the early 1990s, I think the field is really coming back now.”

Gene therapy has had minor successes in very rare diseases but suffered a major setback in 1999 with the death of a patient in a clinical trial at the University of Pennsylvania. Another gene therapy trial treated an immune deficiency but caused cancer in some patients. The general concept of gene therapy — replacing the defective gene in any genetic disease with the intact version — has long been alluring. But carrying it out in practice, usually by loading the replacement gene onto a virus that introduces it into human cells, has been a struggle. The immune system is all too effective at killing the viruses before the genes can take effect.

The success with hemophilia B, reported online Saturday in The New England Journal of Medicine, embodies several minor improvements developed over many years by different groups of researchers. The delivery virus, carrying a good version of the human gene for the clotting agent known as Factor IX, was prepared by researchers at St. Jude Children’s Research Hospital in Memphis. The patients had been recruited and treated with the virus in England by a team led by Dr. Amit C. Nathwani of University College London; researchers at the Children’s Hospital of Philadelphia monitored their immune reactions.

Hemophilia B is caused by a defect in the gene for Factor IX. Fatal if untreated, the disease occurs almost only in men because the Factor IX gene lies on the X chromosome, of which men have only a single copy. Women who carry a defective gene on one X chromosome can compensate with the good copy on their other X chromosome, but they bequeath the defective copy to half their children. About one in 30,000 of newborn boys have the disease, with about 3,000 patients in the United States.

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Old 05-18-2017, 03:34 AM
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Lightbulb Re: Hemophilia & other blood related disorders/diseases

Big step forward...

Human blood stem cells grown in the lab for the first time
17 May 2017 - The stem cells that produce our blood have been created in the lab for the first time. These could one day be used to treat people who have blood diseases and leukaemia with their own cells, rather than bone marrow transplants from a donor. They could also be used to create blood for transfusions.
“This is a very big deal,” says Carolina Guibentif at the University of Cambridge, who was not involved in the research. “If you can develop [these cells] in the lab in a safe way and in high enough numbers, you wouldn’t be dependent on donors.” In a healthy adult, blood stem cells are found in bone marrow, where they replenish the supply of red and white blood cells and platelets. “They are sort of master cells,” says George Daley at Harvard Medical School.

When these cells don’t work properly, they fail to maintain an adequate supply of blood cells. As a result, not enough oxygen reaches the body’s tissues. This can cause serious disease if organs such as the heart are affected. Blood stem cells can also be wiped out by chemotherapy for leukaemia and other cancers. People with these disorders tend to be treated with bone marrow – complete with blood stem cells – from a healthy donor. The difficulty is finding a match. There is a one in four chance of achieving this from a healthy sibling, but the odds are slashed to one in a million if a stranger needs to be found, says Daley.

Making cells

In an attempt to create blood stem cells in the lab, Daley and his colleagues started with human pluripotent stem cells – which have the potential to form almost any other type of body cell. The team then searched for chemicals that might encourage these to become blood stem cells. After studying the genes involved in blood production, the researchers identified proteins that control these genes and applied them to their stem cells.

Potential for a new supply line

They tested many combinations of the proteins, and found five that worked together to encourage their stem cells to become blood stem cells. When they put these into mice, they went on to produce new red and white blood cells and platelets. “It’s very cool,” says Daley. “We’re very excited about the results.” A separate team has achieved the same feat with stem cells taken from adult mice. Raphael Lis at Weill Cornell Medical College in New York and his colleagues started with cells taken from the walls of the animals’ lungs, based on the idea that similar cells in an embryo eventually form the body’s first blood stem cells. The team identified a set of four factors that could encourage these lung stem cells to make them.

Big step forward

Both sets of results represent a “breakthrough”, says Guibentif. “This is something people have been trying to achieve for a long time,” she says. By working with adult mouse epithelial cells, Lis and his team show that the feat could potentially be achieved with cells taken from an adult person. Daley’s team used human stem cells that could in theory be made from skin cells, bolstering the prospect that lab-made human blood could be next. The lab-made stem cells are not quite ready to be used in people just yet, says Daley. Although all of his mice were healthy throughout the experiments, there is a risk that the cells could mutate and cause cancer. And the cells are not quite as efficient at making blood as those found in the body.

But once Daley and his team have honed their procedure, they might be able to make platelets and red blood cells for hospital use. These cell types don’t have a nucleus, so are unable to divide and potentially cause cancer. He hopes this procedure could be used within the next couple of years. Eventually, Daley hopes his cells could be used to create whole blood suitable for transfusions. Not only would such a supply be more reliable than that from donors, but it would also be free of disease. “When new pathogens like Zika pop up, you have to make sure that blood is safe,” says Daley. “We’d be able to have more quality control.”

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