Gene therapy success Story!!!!!!!!
By Jonathan Gitlin
Saturday, June 11, 2005
The biotech revolution has promised much, but often these promises fall short. Gene therapy, which promises to treat diseases by inserting a working copy of a defective gene into a patient, have the potential to revolutionize treatment of genetic disorders. Unfortunately, while the science behind it is sound, often in practice it is difficult to get it to work correctly. The sad case of Jesse Gelsinger, who died following complications related to the adenovirus used to deliver a working version of the OTC gene was a setback in the development of such therapies, and a French study to treat an immunodeficiency used a retrovirus as a vector for gene delivery that disrupted a cancer suppressor gene and caused leukemia in three patients.
Now comes news from a German and Swiss collaboration that have successfully treated patients with chronic granulomatous disease (CGD), an immune deficiency, by removing bone marrow-derived stem cells from the patients, inserting the working copy of the target gene, and then replacing the modified cells. The modified stem cells multiplied successfully and the patients started producing NADPH oxidase. One of the two patients is even able to stop taking preventative antibiotics. This is an encouraging success, although it is still early days for this field.
In other gene therapy news, researchers at the University of Pennsylvania are working on a possible treatment for cystic fibrosis that uses a hybrid virus to deliver a working version of the CTFR gene to the lungs of sufferers of this disease. Controversially, the hybrid virus contains proteins from both HIV and Ebola! The HIV proteins are effective in delivering genes, and the Ebola protein help target the lung epithelial cells. If they gain ethical approval to begin trials in humans the outcome could be very interesting.
article was originally posted on ArsTech
Saturday, June 11, 2005
The biotech revolution has promised much, but often these promises fall short. Gene therapy, which promises to treat diseases by inserting a working copy of a defective gene into a patient, have the potential to revolutionize treatment of genetic disorders. Unfortunately, while the science behind it is sound, often in practice it is difficult to get it to work correctly. The sad case of Jesse Gelsinger, who died following complications related to the adenovirus used to deliver a working version of the OTC gene was a setback in the development of such therapies, and a French study to treat an immunodeficiency used a retrovirus as a vector for gene delivery that disrupted a cancer suppressor gene and caused leukemia in three patients.
Now comes news from a German and Swiss collaboration that have successfully treated patients with chronic granulomatous disease (CGD), an immune deficiency, by removing bone marrow-derived stem cells from the patients, inserting the working copy of the target gene, and then replacing the modified cells. The modified stem cells multiplied successfully and the patients started producing NADPH oxidase. One of the two patients is even able to stop taking preventative antibiotics. This is an encouraging success, although it is still early days for this field.
In other gene therapy news, researchers at the University of Pennsylvania are working on a possible treatment for cystic fibrosis that uses a hybrid virus to deliver a working version of the CTFR gene to the lungs of sufferers of this disease. Controversially, the hybrid virus contains proteins from both HIV and Ebola! The HIV proteins are effective in delivering genes, and the Ebola protein help target the lung epithelial cells. If they gain ethical approval to begin trials in humans the outcome could be very interesting.
article was originally posted on ArsTech
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