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Scientists Cure Hemophiliacs

Scientists Cure Hemophiliacs

Scientist use gene therapy to cure hemophilia…

Scientists say they have successfully cured four patients with Hemophilia – a disease the causes defective blood clotting which leads to excessive bleeding that can kill.

After years of preclinical trials, which including curing hemophiliac mice, scientist finally started trials on humans using gene therapy.

The patients were injected with a specially built virus containing a working version of the gene responsible for blood clotting. The virus implants the gene into liver cells where they begin to produce the chemical need for clotting.

red and white blood cells

Red and White Blood Cells

Image Credit: NCI-Frederick, 2011.

After 6 months of injections to maintain elevated levels of the working gene, four of them were able to stop receiving injections altogether.

The researchers are now monitoring the patients for any signs of liver cancer caused by the virus, a known risk of gene therapy, but so far there have been no complications.

The next stage will be the trial of 20 patients to assess what dosage of the virus is necessary to get enough liver cells making clotting factor so that all patients can stop receiving injections.

The study was published in the New England Journal of Medicine.

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  1. Veronique Greenwood: Four Hemophiliac Patients Successfully Treated with Gene Therapy. Discover Mag, 12/12/2011.

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