title: AAV as a viral vector for human gene therapy. Generation of recombinant virus.
abstract: Investigation of the adeno-associated virus (AAV) life cycle has enabled the establishment of methodology and identification of critical cis-acting sequences required for recombinant AAV production. Vectors derived from the defective human parvovirus (AAV) have been used for successful gene transfer and expression in many diverse mammalian cell types, such as erythroid, airway epithelium, and neuronal cells. One of the crucial steps in the continued case of AAV as a vector is the development of packaging systems that will allow efficient encapsidation of foreign genes into AAV virions. For this reason, the focus of this article will be generation of recombinant AAV vectors.
authors.affiliation: Gene Therapy Center, University of North Carolina at Chapel Hill 27599-7352, USA.
authors.forename: F
authors.initials: F
authors.lastname: Rolling
authors.forename: R J
authors.initials: RJ
authors.lastname: Samulski
mesh_terms: D000818:Animals; D002478:Cells, Cultured / Q000821:virology; D004279:DNA, Viral / Q000235:genetics; D000229:Dependovirus / Q000737:chemistry* / Q000235:genetics*; D004926:Escherichia coli / Q000201:enzymology / Q000235:genetics; D015971:Gene Expression Regulation, Enzymologic; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000737:chemistry*; D016679:Genome, Viral; D006801:Humans; D007668:Kidney / Q000166:cytology / Q000196:embryology / Q000821:virology; D010957:Plasmids; D011401:Promoter Regions, Genetic; D011995:Recombination, Genetic; D017186:Titrimetry; D014162:Transfection; D014779:Virus Replication; D001616:beta-Galactosidase / Q000235:genetics
chemical_list: D004279:DNA, Viral; D001616:beta-Galactosidase