Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV

Academic Article


  • Recombinant adeno-associated virus (rAAV) is a promising vector for retinal application as it transduces photoreceptors and retinal pigment epithelium cells efficiently and in a stable fashion. Because rAAV also transduces retinal ganglion cells, we reasoned that ocular application of rAAV might result in delivery of transgenic protein to the CNS. Here we deSCribe high levels of green fluorescent protein (GFP) persisting at least 6 months in optic nerves and brains of mice and dogs after intravitreal delivery of rAAV-GFP. There was no clinical or histological evidence of inflammatory response although a mild humoral Th-2 response to viral capsid proteins was detected. These findings have important implications with respect to therapeutic applications of rAAV.
  • Authors

    Published In

  • Vision Research  Journal
  • Digital Object Identifier (doi)

    Pubmed Id

  • 3277151
  • Author List

  • Dudus L; Anand V; Acland GM; Chen SJ; Wilson JM; Fisher KJ; Maguire AM; Bennett J
  • Start Page

  • 2545
  • End Page

  • 2553
  • Volume

  • 39
  • Issue

  • 15