title: Adeno-associated virus vector-mediated gene delivery to the vasculature and kidney.
abstract: Relatively successful elsewhere, gene delivery aimed at the vasculature and kidney has made very little progress. In the kidney, the hurdles are related to the unique structure-function relationships of this organ and in the blood vessels to a variety of, mostly endothelial, factors making the delivery of transgenes very difficult. Among gene-therapeutic approaches, most viral gene delivery systems utilized to date have shown significant practical and safety-related limitations due to the level and duration of recombinant transgene expression as well as their induction of a significant host immune response to vector proteins. Recombinant adeno-associated virus (rAAV) vectors appear to offer a vehicle for safe, long-term transgene expression. rAAV-based vectors are characterized by a relative non-immunogenicity and the absence of viral coding sequences. Furthermore, they allow for establishment of long-term latency without deleterious effects on the host cell. This brief review addresses problems related to transgene-delivery to kidney and vasculature with particular attention given to rAAV vectors. The potential for gene therapy as a strategy for selected renal and vascular diseases is also discussed.
authors.affiliation: Division of Nephrology, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL, USA. kaptur@uab.edu
authors.forename: Matthias H
authors.initials: MH
authors.lastname: Kapturczak
authors.forename: Sifeng
authors.initials: S
authors.lastname: Chen
authors.forename: Anupam
authors.initials: A
authors.lastname: Agarwal
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics / Q000502:physiology*; D004730:Endothelium, Vascular / Q000378:metabolism*; D018014:Gene Transfer Techniques; D005822:Genetic Vectors / Q000235:genetics / Q000502:physiology*; D006801:Humans; D007668:Kidney / Q000378:metabolism*