Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a lentiviral vector

Deni S. Galileo, Kristi Hunter, Sylvia B Smith

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Purpose. 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.

Original languageEnglish (US)
Pages (from-to)135-142
Number of pages8
JournalCurrent Eye Research
Volume18
Issue number2
DOIs
StatePublished - Jan 1 1999

Fingerprint

Retinal Pigments
Epithelial Cells
Vitiligo
Microphthalmos
Photoreceptor Cells
Genes
Lac Operon
Complementary DNA
Cell Count
Mutation
Injections

Keywords

  • Gene therapy
  • Lentiviral vector
  • Mitf gene
  • Mouse
  • Retinal degeneration
  • Retinal pigment epithelium (RPE)

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems
  • Cellular and Molecular Neuroscience

Cite this

Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a lentiviral vector. / Galileo, Deni S.; Hunter, Kristi; Smith, Sylvia B.

In: Current Eye Research, Vol. 18, No. 2, 01.01.1999, p. 135-142.

Research output: Contribution to journalArticle

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N2 - Purpose. 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.

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