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AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.
(2017-12-28)
BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia ...
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
(Massachusetts Medical Society, 2020-01-02)
BACKGROUND: Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration ...