How Does Gene Therapy Treat Hemophilia?

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Scientists have been testing new gene therapies to treat hemophilia, an inherited blood disorder. Different hemophilia trails have been done all over the world. Researchers have made some breakthroughs in this field. The aim of gene therapy is to make enough clotting factors while protecting the immune system from destroying. In one trial, 13 patients received a single infusion of an nvestigational gene therapy called valoctocogene roxaparvovec. After the trail, 11 of which succeeded in achieving normal or almost near factor VIII levels.   Keywords: gene therapy hemophilia; hemophilia gene therapy