[Gene therapy in ophthalmology].
Replication-deficient adenoviral vectors have been used to transfer foreign DNA into a variety of cells including 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 and are potential targets for gene therapy. We have therefore analysed the ability of an adenoviral vector to transfer in vivo the E. coli LacZ 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, the anterior chamber or the peribulbar space did not result in any detectable cytopathic effect and was associated with endocytosis of viral particles in corneal endothelial, photoreceptor, bipolar, ganglionic and oculomotor muscle cells, depending on the administration route. At the viral titer used (3 10(7) or 10(9) pfu), the expression was detected for at least 50 days. These results open new prospects for the treatment of some retinal hereditary disorders and acquired corneal or retinal alterations due to inflammatory disease.
Mashhour
B
B
INSERM U 380, ICGM, Paris.
D000260:Adenoviruses, Human / Q000235:genetics*; D000818:Animals; D005128:Eye Diseases / Q000628:therapy*; D015316:Genetic Therapy; D005822:Genetic Vectors; D051379:Mice; D008810:Mice, Inbred C57BL; D011817:Rabbits
Thérapie génique en ophtalmologie.