Novel gene therapy may lead to cure in hemophilia A patients: Even hard to treat subset requiring expensive treatment

Source: Medical College of Wisconsin
Posted: July 3, 2006

Summary:

A discovery by Medical College of Wisconsin and BloodCenter of Wisconsin researchers in Milwaukee may be a key to a permanent genetic cure for hemophilia A patients, including a subset who do not respond to conventional blood transfusions. A non-replicative virus containing the FVIII gene would be introduced into the stem cells from the patient. The FVIII engineered for production only in platelets would insert itself into the DNA of the stem cells. These same stem cells would then be given back to the donor patient and the stem cells would continue to make blood cells normally, releasing the life saving FVIII only when the platelets stick to a bleeding site of injury. Researchers believe the blood cells could be continually produced throughout a patient's life span, and treatment could normalize bleeding for patients with hemophilia.

Commentary: Maybe this research could lead to effective treatments, or even a cure for hemophilia.