Gene therapy with chimeric oligonucleotides delivered by a method comprising a step of iontophoresis
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United States of America Patent
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app pub date -
Oct 18, 2004
filing date -
Apr 17, 2001
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Abandoned
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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.
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