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Gene Therapy for Hemophilics
 
   

Use of Implants in the treatment of Hemophilia

Scientists have shown that a type of implant is capable of producing a protein that is used for the treatment of Haemophilia.

Thumb size implants were placed in monkeys and guinea pigs. These implants converted a natural protein in the animals to a new form of protein called factor VIIa. This protein is used in the treatment of haemophilia.

Hemophilia is caused by the lack of a clot-promoting protein called Factor VIII. Patients can be treated with infusions of that protein. But some 15 percent of such patients develop antibodies that inactivate the infused protein. They can be treated with infusions of Factor VIIa instead, but that is expensive and the protein disintegrates quickly.

The implants could avoid both those drawbacks, as well as the antibody problem, the researchers said.

The implants are porous chambers containing a substance that converts Factor VII to Factor VIIa. The monkey, which had three implants in its abdomen, generated Factor VIIa for a month before the implants were removed for inspection.

The work was reported in the March issue of the journal Nature Biotechnology by Dr. Harvey Pollard of the Uniformed Services University School of Medicine in Bethesda, Md., and colleagues.

   



 
 
 
 
 
             
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