• One-dose gene therapy produces clotting factor, safely stops bleeding in hemophilia B patients

    7 days ago - By ScienceDaily

    A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy enabled adult participants to safely produce sustained levels of clotting factor that prevented debilitating bleeding episodes. Patients were able to terminate prophylactic treatments; the gene therapy nearly universally eliminated their need for intravenous infusions of manufactured clotting factor.
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  • Gene Therapy May Allow Hemophilia Patients to Go without Meds

    Gene Therapy May Allow Hemophilia Patients to Go without Meds

    7 days ago - By MedlinePlus

    Related MedlinePlus Pages: Genes and Gene Therapy , Hemophilia
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  • Gene Therapy May Let Hemophilia Patients Skip Meds

    7 days ago - By Web MD

    This could eliminate the need for tedious and costly standard treatments, researchers report.
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