In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium

Gene Ther. 2001 Nov;8(21):1665-8. doi: 10.1038/


We have evaluated the transduction profiles of an HIV-based lentiviral vector delivered regionally to ocular tissues in vivo. Following subretinal injection, a green fluorescent protein (GFP) reporter gene was efficiently and stably expressed in retinal pigment epithelial (RPE) cells. Limited transduction of adjacent photoreceptors occurred in newborn mice, but was inefficient in adult animals. Injection of the vector into the anterior chamber resulted in efficient and stable transduction of corneal endothelial cells. Efficient in vivo gene transfer into cells of the corneal endothelium and retinal pigment epithelium by lentiviral vectors may therefore offer a valuable approach to the treatment of disorders of the cornea and outer retina.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Animals, Newborn
  • Epithelium, Corneal / metabolism
  • Eye / metabolism*
  • Eye Diseases / therapy
  • Gene Expression
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage*
  • Green Fluorescent Proteins
  • HIV-1 / genetics*
  • Luminescent Proteins / genetics
  • Mice
  • Mice, Inbred C57BL
  • Microinjections
  • Pigment Epithelium of Eye / metabolism
  • Transduction, Genetic / methods*


  • Luminescent Proteins
  • Green Fluorescent Proteins