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to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)

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Offline 7assan

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to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)
« on: January 14, 2010, 03:26:14 PM »
β-Thalassemia Treatment Succeeds, With a Caveat

Gene therapy researchers are approaching a key milestone: They appear to have controlled an inherited blood disorder called β-thalassemia that's more common than any disease treated so far with gene therapy. A young man who received new genes to repair blood cells no longer needs regular transfusions and, 2 years later, seems healthy. Describing this success last week to a U.S. review panel in Bethesda, Maryland, a researcher added a caveat: The inserted gene may have turned on growth signals, raising the potential for cancer.

http://www.sciencemag.org/cgi/content/summary/326/5959/1468-b
« Last Edit: January 15, 2010, 07:41:05 AM by baloch »
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Offline suk838

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Re: to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)
« Reply #1 on: January 15, 2010, 07:04:26 AM »
good news thanks alot.  :wink
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Offline 7assan

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Gene therapy for beta-thalassemia successful in mice
« Reply #2 on: January 15, 2010, 07:43:45 AM »
More than 200 million people around the world suffer from a group of inherited blood disorders called thalassemias, which, in severe forms, are fatal. These diseases are caused by the faulty production of hemoglobin, the protein in a red blood cell that transports oxygen to the tissues in the body. Now, a research team based at the Memorial Sloan-Kettering Cancer Center in New York has used a gene to bring one form of the disease, beta-thalassemia, under control in mice. The result offers hope that the same approach may work in people.

Hemoglobin is made up of four pieces: two beta-globin and two alpha-globin proteins. In beta-thalassemia, the problem is the production of beta-globin parts. When only one copy of the beta-globin gene is damaged, the result is a mild anemia that does not require treatment. But when both copies are damaged, children often die at a young age. Severe beta-thalassemia is treated with regular blood transfusions, and, in some cases, a bone marrow transplant. But bone marrow transplants are not available to everyone, and are risky because of complications with the immune system. Transfusions can cause a dangerous build-up of iron in the heart muscle.


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Both transfusions and transplants have risks
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"That is what makes a genetic approach so attractive," says Michel Sadelain, a gene therapy researcher at the Memorial Sloan-Kettering Cancer Center. If a patient's own bone marrow cells are removed and have a healthy beta-globin gene inserted, the cells are returned to the patient and normal hemoglobin should be produced without any complications with the immune system, he says. The team tested this approach in mice. The results are presented in a recent issue of Nature.

Sadelain's group packaged the healthy beta-globin gene and an adjacent area of the DNA, which controls when and where the gene is turned on, into a virus. The virus was then mixed with bone marrow cells that had been removed from mice with beta-thalassemia. When the virus injects the DNA into the cell, the gene joins the rest of the DNA and becomes part of the cell's chromosomes. When this happens to bone marrow stem cells, the precursors for all the blood cells in the body, the healthy beta-globin gene will be passed to all future generations of blood cells, says Sadelain.

Fifteen weeks after the altered bone marrow cells were placed back into the mouse, hemoglobin levels had risen to 12-13 grams per deciliter (g/dL) compared to 8-9 g/dl in untreated mice with an intermediate form of beta-thalassemia. Healthy mice have hemoglobin levels in the range of 15 g/dl.

The production of normal hemoglobin from the newly introduced gene corrected the sickly appearance of the red blood cells. Normal red blood cells are packed with hemoglobin and are bright, plump, and round with a dimple in the center. "The red blood cells from a person or mouse with beta-thalassemia have very low levels of hemoglobin. The cells are pale and fragile and have bizarre shapes like tear drops and spikes, which are easy to recognize under the microscope," says Sadelain.


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Curative levels of hemoglobin were produced in mice
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While other groups have tried to insert a healthy beta-globin gene into mice, none have succeeded in getting curative levels of the hemoglobin protein. Until now, it has also been difficult to restrict the production to only the red blood cells, according to Charles Peterson, Director of the Blood Diseases Program at the National Heart, Lung, and Blood Institute in Bethesda, Maryland. High levels of hemoglobin are toxic to other cell types.

"Sadelain's group gets 17 to 24 percent production of the healthy beta-globin protein, which is excellent, because with this level you fix most of what goes wrong," says Peterson.

The work also holds promise for sickle cell anemia, which also results from two copies of a faulty beta-globin gene, and which might be treated the same way, says Peterson. About one in 12 people of African descent worldwide has a single copy of the sickle cell gene, while one in 400 has the disease.

First discovered in people living around the Mediterranean Sea, Thalassemia gets its name from the Greek "thalassa" meaning sea and "emia" meaning blood. The disease is particularly prevalent among Greeks and Italians, Southeast Asians and Africans. The sickle cell and beta-thalassemia genes are thought to have persisted in these populations because having a copy of either gene protects against malaria.




http://www.thestar.com/News/GTA/article/261446
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Offline Sharmin

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Re: to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)
« Reply #3 on: January 15, 2010, 03:44:00 PM »
Praying for this with my whole heart and soul  :pray :pray :pray :pray :pray :pray :pray

For my little guy and for all of you  :grouphug

Sharmin
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Offline Andy Battaglia

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Re: to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)
« Reply #4 on: January 15, 2010, 05:07:00 PM »
If anyone can actually find any text from the above article, I would very much like to see it, as this has no details at all. I would like to know which trial this refers to and what the evidence is, but the article is a pay article.

One has to be realistic about all new treatments and cures, as far as a time line for approval. Gene therapy will not be available overnight, no matter how well any trial goes. Meanwhile, please do realize that with the treatment available today, thalassemia is a very manageable disorder as long as patients comply fully with their treatment. This will only continue to improve in the coming years as we wait for this monumental development in gene therapy.
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Andy

All we are saying is give thals a chance.
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Offline 7assan

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Re: to dear andy (Gene Therapy: β-Thalassemia Treatment Succeeds, With a Caveat)
« Reply #5 on: January 15, 2010, 05:34:27 PM »
Thnx dear Andy For ur reply
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