Plant-made Hemophilia Therapy Shows Promise, Penn Study Finds

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People with hemophilia require regular infusions of clotting factor to prevent them from experiencing uncontrolled bleeding. But a significant fraction develop antibodies against the clotting factor, essentially experiencing an allergic reaction to the very treatment that can prolong their lives.

People with hemophilia require regular infusions of clotting factor to prevent them from experiencing uncontrolled bleeding. But a significant fraction develop antibodies against the clotting factor, essentially experiencing an allergic reaction to the very treatment that can prolong their lives.

Researchers from the University of Pennsylvania School of Dental Medicine and University of Florida have worked to develop a therapy to prevent these antibodies from developing, using a protein drug produced in plant cells to teach the body to tolerate rather than block the clotting factor.

Successful results from a new study of the treatment in dogs give hope for an eventual human treatment.

Henry Daniell, a professor in Penn Dental Medicine’s Department of Biochemistry and director of translational research, was the senior author on the study, collaborating on the work with his former advisee, Roland W. Herzog, a professor at the University of Florida and lead author on the paper. The work was published in the journal Molecular Therapy.

Read more at University of Pennsylvania

Photo credit: Henry Daniell, University of Pennsylvania