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Ancient DNA helps UF researchers unearth potential hemophilia therapy

nse by performing gene therapy on mice within 24 hours of their birth, when their immune systems were still naive and would accept the protein.

This type of approach cannot be used in human babies yet because doctors have no way of gauging how severe hemophilia is in newborns. Patients with mild disease will have fewer problems and the benefits of gene therapy may not outweigh risks to the baby, Fletcher said.

Ponder said she thinks the technique also may prove to share some of the same problems as viral-based gene therapy, potentially activating cells that cause cancer.

Now UF researchers are studying different ways to use the transposon and trying to find a way to overcome the immune attack when performing gene therapy on adult animals.

"I don't think the research is done," Mah said. "But this is definitely a step forward for hemophilia gene therapy."


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Source:University of Florida


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