A team of biologists and chemists has developed a technique that
promises to head off hemophilia before birth.
Led by Elliot D. Rosen, associate director of Notre Dame's W.M.
Keck Center for Transgene Research, researchers from the Keck
Center and ND's Department of Chemistry and Biochemistry succeeded
in transferring liver cells into mouse embryos that lacked the
ability to produce an essential blood-clotting protein.
Mice unable to produce the protein either die before birth or
shortly thereafter. But those receiving the cell transplant survived
long term, with the new cells living on in the livers of about
half of the adult mice.
The technique could become a new pre-birth way to address human
hemophilia, a group of hereditary bleeding disorders. Hemophiliacs
fail to produce enough blood-clotting proteins, resulting in spontaneous
bleeding, particularly in joints, muscles and internal organs.
Conventional hemophilia treatments rely on injections of a substitute
coagulation factor obtained from donor plasma or, more recently,
genetically engineered preparations. But more than 15 percent
of patients develop antibodies to the substitutes and become resistant
to further treatment. The factors produced by the transplanted
cells don't appear to trigger an immune reaction.
The researchers described the transplant technique in a paper
published earlier this year in the inaugural issue of the Journal
of Thrombosis and Haemostasis.
