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  • Title: Receptor targeting of adeno-associated virus vectors.
    Author: Büning H, Ried MU, Perabo L, Gerner FM, Huttner NA, Enssle J, Hallek M.
    Journal: Gene Ther; 2003 Jul; 10(14):1142-51. PubMed ID: 12833123.
    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.
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