Receptor targeting of adeno-associated virus vectors.
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.
Büning
H
H
Genzentrum Ludwig-Maximilians-Universität München, Feodor-Lynen-Strasse 25, 81377 Münich, Germany.
Ried
M U
MU
Perabo
L
L
Gerner
F M
FM
Huttner
N A
NA
Enssle
J
J
Hallek
M
M
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
D000906:Antibodies; D036022:Capsid Proteins; D008024:Ligands; D011956:Receptors, Cell Surface