Abstract: The present invention provides a device and a method for the enhanced retinal delivery of nucleic acid therapeutics utilizing iontophoresis to evoke a transient elongation of the Müller Cells of a mammalian eye. The enhanced retinal deposition can be achieved by either a topical application, subconjunctival, or an intravitreal injection of the nucleic acid composition followed by, preceded by, or administered simultaneously with the iontophoretic application. The present invention thus provides a particularly advantageous method for the treatment of ocular diseases comprising the in vivo administration of a nucleic acid capable of alleviating the symptoms of a disease, the delivery of the nucleic acid being enhanced by using iontophoresis. This method can be applied particularly to the diseases of the retina resulting from an alteration of a gene expression and/or the over-expression of particular growth factors.

Abstract: The present invention provides a method for enhancing the in vivo delivery of chimeric oligonucleotides, containing for example DNA/2?OMeRNA, into cells of a plant, an animal or a human, comprising a step of applying topically to or injecting into a tissue, or tissue adjacent to a tissue, containing said cells, a composition comprising said chimeric oligonucleotide, followed by, preceded by, or simultaneously to a step of transferring said chimeric oligonucleotide into said cells by iontophoresis, and relates to a gene therapy method comprising the iontophorically transfer of a chimeric oligonucleotide DNA/2?OMeRNA. The present invention is also directed to particular chimeric oligonucleotides DNA/2?OMeRNA capable of inducing or inhibiting the expression of a specific gene involved in eye function by inducing or reverting a mutation in that specific gene, and their use as therapeutic composition for preventing or treating ocular diseases.

Abstract: The present invention provides a method for enhancing the in vivo delivery of chimeric oligonucleotides, containing for example DNA/2′OMeRNA, into cells of a plant, an animal or a human, comprising a step of applying topically to or injecting into a tissue, or tissue adjacent to a tissue, containing said cells, a composition comprising said chimeric oligonucleotide, followed by, preceded by, or simultaneously to a step of transferring said chimeric oligonucleotide into said cells by iontophoresis, and relates to a gene therapy method comprising the iontophorically transfer of a chimeric oligonucleotide DNA/2′OMeRNA. The present invention is also directed to particular chimeric oligonucleotides DNA/2′OMeRNA capable of inducing or inhibiting the expression of a specific gene involved in eye function by inducing or reverting a mutation in that specific gene, and their use as therapeutic composition for preventing or treating ocular diseases.