title: Adenoviral Vectors for Hemophilia Gene Therapy.
abstract: Hemophilia is an inherited blood clotting disorder resulting from deficiency of blood coagulation factors. Current standard of care for hemophilia patients is frequent intravenous infusions of the missing coagulation factor. Gene therapy for hemophilia involves the introduction of a normal copy of the deficient coagulation factor gene thereby potentially offering a definitive cure for the bleeding disorder. A variety of approaches have been pursued for hemophilia gene therapy and this review article focuses on those that use adenoviral vectors.
authors.affiliation: Telethon Institute of Genetics and Medicine, Naples, Italy ; Department of Translational Medicine, Federico II University of Naples, Italy.
authors.forename: N
authors.initials: N
authors.lastname: Brunetti-Pierri
authors.affiliation: Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
authors.forename: Philip
authors.initials: P
authors.lastname: Ng
keywords: Adenovirus; Factor IX; Factor VIII; Gene therapy; Helper-dependent adenoviral vectors; Hemophilia A; Hemophilia B; Viral vectors