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Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a
D S Galileo1, K Hunter, S B Smith
1Department of Cellular Biology and Anatomy, Medical College of Georgia, Augusta 30912-2000, USA. dgalileo@mail.mcg.edu
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.