Sunday, November 6, 2011

Gene therapy shows promise as hemophilia treatment in animal studies

          Scientists have successfully found a way to dramatically lessen the severity of hemophilia A in large animals.  The process involves inserting a specific gene for clotting factor VIII into engineered adult stem cells, and then injecting these cells into the abdominal cavity of the animal.  Studies showed that the cells migrated to the animals' joints and reversed the bleeding and damage, restoring proper posture and walking ability.  All other spontaneous bleeding was also stopped.  However, the subjects somehow developed an immune response to clotting factor VIII, suggesting that the treatment's effects may be shorter-lived than hoped.  If this problem can be solved, this procedure would be a milestone in treating and effectively curing the disease in humans.

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