Ds. Galileo et al., Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a lentiviral vector, CURR EYE R, 18(2), 1999, pp. 135-142
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 pro
moter would stably infect a significant number of RPE cells in the vitiligo
mouse. This mouse harbors a mutation in the microphthalmia gene in RPE cel
ls that leads to slow progressive photoreceptor cell degeneration.
Methods. Concentrated lentiviral vector HR'CMVlacZ was injected intravitrea
lly 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 fo
r a period of at least two months. These results raise the possibility of u
sing a similar lentiviral vector for introduction of a correct copy of the
microphthalmia cDNA into the RPE that may ultimately rescue photoreceptor c
ells in this mutant mouse.