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Prenatal Gene Transfer Proposed For Treating Hemophilia, Other Diseases.
2002 FEB 14 - (NewsRx.com & NewsRx.net) -- by Sonia Nichols, senior medical writer - Based on the results of animal model experiments, a research team in Europe has proposed that gene transfer could be used for treating certain diseases in infants before they are even born.
The group, headed by Holm Schneider of the Children's Hospital at the University of Erlangen-Nuernberg, Germany, used virus-based gene therapy for treating hemophilia in mouse fetuses. Hemophilia is an inherited disease whereby a patient does not produce clotting factors that could result in life-threatening hemorrhage.
Schneider's group has suggested that fetuses may have the ability to take up genes that can stimulate the production of clotting factors better than infants can once they are born. In addition, their immune systems are immature, making them less likely to form problematic antibodies often seen in older patients with hemophilia.
The researchers used two different types of viral vectors to transfer human clotting factor IX (hfIX) DNA to late-stage murine fetuses via their muscles, abdominal cavities, or vascular systems while they remained in their mothers' uteri.
"Mice treated in utero by intramuscular injection of an adenoviral vector carrying hfIX cDNA exhibited high-level gene expression at birth and therapeutic - albeit continuously decreasing - plasma concentrations of hfIX over the entire 6 months of the study," Schneider and coworkers reported.
Laboratory examination of several fetal mice showed that viruses containing the clotting factor genes disseminated throughout many organs soon after the gene therapy was administered.
Another virus, adeno-associated virus, stimulated the production of clotting factor also, but levels were lower ...
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Source: HighBeam Research, Prenatal Gene Transfer Proposed For Treating Hemophilia, Other...