For the treatment of hemophilia B there is gene therapy. Studies with adults are already underway. With this curative therapeutic approach, the missing or faulty gene is replaced. A healthy factor IX gene is introduced into the liver cell via a virus vector. “The gene is not integrated into our genome, it is only there in the cell nucleus,” says Kramer. There it is read off so that the liver cell can produce the healthy factor IX itself. The result: Even after a single infusion, the adult test subjects with severe haemophilia exhibited such good factor IX activity that they either no longer had to inject at all or only had to inject in everyday life if they were seriously injured or operations were necessary . In the foreseeable future, gene therapy will also be available for the treatment of haemophilia A patients, says Kramer. Studies are already underway here too. However, the factor VIII gene is more complicated in gene therapy than the factor IX gene. Hence the delay.
In Regensburg: gene therapy for children for the first time
As one of the few centers in Germany, the University Hospital Regensburg is now offering hemophilia B therapy for the first time as part of a study for children and adolescents under 18 years of age. Life with the disease should also be made easier for them – initially for patients between the ages of twelve and 17 in whom the residual factor IX activity is a maximum of two percent, then for boys between four and eleven and finally for toddlers under four years of age.
Inquiries from kindergartens and schools about what sick children are allowed to and what not, as well as the anxiety and uncertainty of the caregivers show that hemophilia leads to exclusion relatively quickly. You can usually counteract this with good educational talks. “The children are allowed to be children in everyday life,” says Kramer. “But ultimately we advise against certain high-risk sports, such as martial arts, ball sports, and contact sports of any kind, which can lead to increased injuries.”
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