title: Parvovirus-mediated gene transfer for the haemophilias.
abstract: Gene therapy may revolutionize the treatment of haemophilia. Effective gene therapy requires sustained therapeutic levels of factors IX (FIX) and VIII. Adeno-associated virus (AAV) is a member of the parvovirus family, is a nonpathogenic virus with a broad host cell range, and does not provoke a significant immune response upon infection. These favourable characteristics make AAV a suitable gene transfer vector for factor deficient patients. A new understanding of AAV biology coupled with novel AAV vector designs suggest that the goal of effective gene transfer is within reach. We review here recent advances in AAV vectors used for gene transfer of the haemophilias.
authors.affiliation: UNC Gene Therapy Center, University of North Carolina, Chapel Hill NC, USA. cwalsh@med.unc.edu
authors.forename: C E
authors.initials: CE
authors.lastname: Walsh
authors.forename: H
authors.initials: H
authors.lastname: Chao
mesh_terms: D015153:Blotting, Western; D000229:Dependovirus / Q000235:genetics*; D005164:Factor IX / Q000235:genetics*; D005169:Factor VIII / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D012703:Serotyping
chemical_list: D005169:Factor VIII; D005164:Factor IX