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Updated: Mar 23, 2026

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
Published on: February 2, 2018
Gene Therapy for Hemophilia
Arthur W Nienhuis1, Amit C Nathwani2, Andrew M Davidoff3
11 Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital , Memphis, Tennessee.
Abstract:
Adeno-associated viral vectors have been developed for the treatment of hemophilia A and B. Derivation of vector particles is achieved after multiplasmid transfection of cells that package the vector genome to yield vector particles. To date, three clinical trials have been performed for hemophilia B. The results of these trials are described. The trial that we conducted with our collaborators has yielded evidence of clinical efficacy for hemophilia B. A vector for treating hemophilia A has been developed and a clinical trial is planned.
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