title: Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a lentiviral vector.
abstract: OBJECTIVE
  The purpose of the present study was to test whether a lentiviral vector encoding the marker lacZ gene under the control of the human CMV promoter would stably infect a significant number of RPE cells in the vitiligo mouse. This mouse harbors a mutation in the microphthalmia gene in RPE cells that leads to slow progressive photoreceptor cell degeneration.
  
  
  METHODS
  Concentrated lentiviral vector HR'CMVlacZ was injected intravitreally into newborn vitiligo mice. Mice were sacrificed at various time points up to two months post-injection and eyes were processed histochemically to detect lacZ expression.
  
  
  RESULTS
  The lentiviral vector infected predominantly the RPE and resulted in lacZ expression in numerous RPE cells at all times analyzed.
  
  
  CONCLUSIONS
  LacZ expression in vitiligo RPE cells appeared to be stable for a period of at least two months. These results raise the possibility of using a similar lentiviral vector for introduction of a correct copy of the microphthalmia cDNA into the RPE that may ultimately rescue photoreceptor cells in this mutant mouse.
authors.affiliation: Department of Cellular Biology and Anatomy, Medical College of Georgia, Augusta 30912-2000, USA. dgalileo@mail.mcg.edu
authors.forename: D S
authors.initials: DS
authors.lastname: Galileo
authors.forename: K
authors.initials: K
authors.lastname: Hunter
authors.forename: S B
authors.initials: SB
authors.lastname: Smith
mesh_terms: D000818:Animals; D000831:Animals, Newborn; D004268:DNA-Binding Proteins / Q000235:genetics*; D018014:Gene Transfer Techniques; D005822:Genetic Vectors; D007763:Lac Operon / Q000235:genetics; D016086:Lentivirus / Q000502:physiology*; D051379:Mice; D008810:Mice, Inbred C57BL; D008817:Mice, Mutant Strains; D051739:Microphthalmia-Associated Transcription Factor; D010857:Pigment Epithelium of Eye / Q000473:pathology / Q000821:virology*; D012162:Retinal Degeneration / Q000235:genetics / Q000473:pathology; D014157:Transcription Factors / Q000235:genetics*
chemical_list: D004268:DNA-Binding Proteins; D051739:Microphthalmia-Associated Transcription Factor; C497235:Mitf protein, mouse; D014157:Transcription Factors