Ns. Dejneka et al., Pharmacologically regulated gene expression in the retina following transduction with viral vectors, GENE THER, 8(6), 2001, pp. 442-446
The availability of inducible expression systems makes regulatable control
of therapeutic proteins an attainable goal in gene therapy. We delivered te
tracycline-inducible transgenes to the subretinal space using recombinant a
denoviruses. Upon administration of doxycycline, we demonstrated reversible
expression of green fluorescent protein in the retinal pigment epithelium
as well as modulation of human growth hormone produced in the retina and se
creted in the blood stream. This mode of delivery and regulation offers a u
nique way to evaluate gene function in the eye and represents a novel metho
d for introducing therapeutic proteins into the retina.