GENE-THERAPY IN OCULAR-TISSUES

Replication-deficient adenoviral vectors have been used to transfer fo reign DNA into a variety of cells inducing post-mitotic cells, in vivo . They constitute the obligatory targets of gene transfer for a number of ocular diseases that have been elucidated at the molecular level a nd are potential targets for gene therapy. We have therefore analysed the ability of an adenoviral vector to transfer in vivo the E. coil La cZ gene into ocular cells of mice and rabbits. Injection of up to 3 10 (7) pfu in mice and 10(9) pfu in rabbits, into the vitreous cavity, th e anterior chamber or the peribulbar space did not result in any detec table cytopathic effect and was associated with endocytosis of viral p articles in corneal endothelial, photoreceptor, bipolar, ganglionic an d oculomoteur muscle cells, depending on the administration route. At the viral titer used (3 10(7) or 10(9) pfu), the expression was detect ed for at least 50 days. These results open new prospects for the trea tment of some retinal hereditary disorders and acquired corneal or ret inal alterations due to inflammatory disease.