title: Gene Therapy for Hemophilia.
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.
authors.affiliation: 1 Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital , Memphis, Tennessee.
authors.forename: Arthur W
authors.initials: AW
authors.lastname: Nienhuis
authors.affiliation: 2 Department of Haematology, University College London Cancer Institute , London, United Kingdom .
authors.forename: Amit C
authors.initials: AC
authors.lastname: Nathwani
authors.affiliation: 3 Department of Surgery, St. Jude Children's Research Hospital , Memphis, Tennessee.
authors.forename: Andrew M
authors.initials: AM
authors.lastname: Davidoff
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000378:metabolism; D015316:Genetic Therapy; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans