title: Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral Approach.
abstract: OBJECTIVE
  Gene therapy involves delivery of exogenous DNA to provide a therapeutic protein. Ideally, a gene therapy vector should be non-toxic, non-immunogenic, easy to produce, and efficient in protecting and delivering DNA into target cells.
  
  
  METHODS
  Adeno-associated virus (AAV) offers these advantages and few, if any, disadvantages, and over 100 isolates exist. We previously showed that AAV-mediated gene therapy can be used to restore vision to patients with Leber's congenital amaurosis, a disease of childhood blindness.
  
  
  RESULTS
  Here we show that novel recombinant AAV2/8 and AAV2/9 transduce kidney tubule cells with high efficiency both in vitroin cell culture and in vivoin mice. In addition, we adapted and modified a retrograde approach to allow for optimal transgene delivery to renal tubular cells that further minimizes the risk of an immunogenic reaction.
  
  
  CONCLUSIONS
  We believe that recombinant AAV2, especially AAV2/8, gene delivery to renal tubule cells via a retrograde approach represents a viable method for gene therapy for a multitude of renal disorders ranging from autosomal dominant polycystic kidney disease to acute kidney injury.
authors.affiliation: Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pa., USA.
authors.forename: Daniel C
authors.initials: DC
authors.lastname: Chung
authors.forename: Ben
authors.initials: B
authors.lastname: Fogelgren
authors.forename: Kwon Moo
authors.initials: KM
authors.lastname: Park
authors.forename: Jessica
authors.initials: J
authors.lastname: Heidenberg
authors.forename: Xiaofeng
authors.initials: X
authors.lastname: Zuo
authors.forename: Liwei
authors.initials: L
authors.lastname: Huang
authors.forename: Jean
authors.initials: J
authors.lastname: Bennett
authors.forename: Joshua H
authors.initials: JH
authors.lastname: Lipschutz