发明名称 GENE THERAPY WITH CHIMERIC OLIGONUCLEOTIDES DELIVERED BY A METHOD COMPRISING A STEP OF IONTOPHORESIS
摘要 <p>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 an 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, preceded by, or simultaneous 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/'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.</p>
申请公布号 WO2002083184(A2) 申请公布日期 2002.10.24
申请号 IB2002002310 申请日期 2002.04.17
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