title: Receptor targeting of adeno-associated virus vectors.
abstract: Adeno-associated virus (AAV) is a promising vector for human somatic gene therapy. However, its broad host range is a disadvantage for in vivo gene therapy, because it does not allow the selective tissue- or organ-restricted transduction required to enhance the safety and efficiency of the gene transfer. Therefore, increasing efforts are being made to target AAV-2-based vectors to specific receptors. The studies summarized in this review show that it is possible to target AAV-2 to a specific cell. So far, the most promising approach is the genetic modification of the viral capsid. However, the currently available AAV-2 targeting vectors need to be improved with regard to the elimination of the wild-type AAV-2 tropism and the improvement of infectious titers. The creation of highly efficient AAV-2 targeting vectors will also require a better understanding of the transmembrane and intracellular processing of this virus.
authors.affiliation: Genzentrum Ludwig-Maximilians-Universität München, Feodor-Lynen-Strasse 25, 81377 Münich, Germany.
authors.forename: H
authors.initials: H
authors.lastname: Büning
authors.forename: M U
authors.initials: MU
authors.lastname: Ried
authors.forename: L
authors.initials: L
authors.lastname: Perabo
authors.forename: F M
authors.initials: FM
authors.lastname: Gerner
authors.forename: N A
authors.initials: NA
authors.lastname: Huttner
authors.forename: J
authors.initials: J
authors.lastname: Enssle
authors.forename: M
authors.initials: M
authors.lastname: Hallek
mesh_terms: D000818:Animals; D000906:Antibodies / Q000235:genetics; D036022:Capsid Proteins / Q000235:genetics; D000229:Dependovirus / Q000235:genetics*; D018390:Gene Targeting / Q000379:methods*; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000235:genetics; D016679:Genome, Viral; D006801:Humans; D008024:Ligands; D011956:Receptors, Cell Surface / Q000378:metabolism*; D014161:Transduction, Genetic / Q000379:methods
chemical_list: D000906:Antibodies; D036022:Capsid Proteins; D008024:Ligands; D011956:Receptors, Cell Surface