首页> 外国专利> Method for releasing in vivo a chimeric oligonucleotide into target cells of a human or animal tissue, to treat a disease, to obtain an animal model, to select cosmetic or pharmaceutical compounds, to treat a human host having retinopathy and to treat a host human or animal having ocular neovascularization, chimeric alligonucleotide, pharmaceutical composition, animal model, and use thereof

Method for releasing in vivo a chimeric oligonucleotide into target cells of a human or animal tissue, to treat a disease, to obtain an animal model, to select cosmetic or pharmaceutical compounds, to treat a human host having retinopathy and to treat a host human or animal having ocular neovascularization, chimeric alligonucleotide, pharmaceutical composition, animal model, and use thereof

机译:在体内将嵌合寡核苷酸释放到人或动物组织的靶细胞中以治疗疾病,获得动物模型,选择化妆品或药物化合物,治疗患有视网膜病的人宿主以及治疗人或动物宿主的方法具有眼新血管形成的动物,嵌合的寡核苷酸,药物组合物,动物模型及其用途

摘要

"METHODS TO RELEASE A CHEMICAL OLIGONUCLEOTIDE INSIDE TARGET CELLS OF A HUMAN OR ANIMAL TISSUE, TO TREAT A DISEASE, TO OBTAIN AN ANIMAL MODEL, TO SELECT COSMETIC COMPOUNDS AND A POST HETARIAN PHARMACEUTICAL COMPOUNDS HUMAN OR ANIMAL HOSTING WITH OCCULAR NEOVASCULARIZATION, CHEMICAL OLIGONUCLEOTIDE, PHARMACEUTICAL COMPOSITION, ANIMAL MODEL, AND, USE OF THE SAME ". The present invention provides a method for enhancing the in vivo release of chimeric oligonucleotides, for example containing DNA / 2α OMeRNA, into cells of a plant, animal, or human, comprising a step of topically applying to or injecting into a tissue, or tissue adjacent to a tissue, containing said cells, a composition comprising said chimeric oligonucleotide, then preceded by or simultaneous with a step of iontophoresis said chimeric oligonucleotide into said cells, and refers to a method of gene therapy comprising iontophoresis transfer of a chimeric DNA / 2? OMeRNA oligonucleotide. The present invention also relates to particular chimeric DNA / 2? OMeRNA oligonucleotides capable of inducing or inhibiting the expression of a specific gene involved in ocular function by inducing or retaining a mutation in that specific gene, and its use as a therapeutic composition for prevent or treat eye diseases.
机译:“在人或动物组织的靶细胞内释放化学寡核苷酸的方法,以治疗疾病,获得动物模型,选择化妆品化合物和后希他性的药物化合物,例如由核或类固醇组成的,呈球形或球形的类固醇,呈环状或球形成分,动物模型以及相同的用法。本发明提供了一种增强嵌合寡核苷酸(例如含有DNA /2αOMeRNA)在体内释放到植物,动物或人的细胞中的方法,该方法包括局部施用于或注射入组织或组织的步骤。邻近包含所述细胞的组织的组合物,其包含所述嵌合寡核苷酸,然后在将所述嵌合寡核苷酸电渗入所述细胞之前或与之同时进行离子电渗步骤,并且涉及基因治疗方法,其包括将嵌合DNA / 2离子电渗的方法? OMeRNA寡核苷酸。本发明还涉及特定的嵌合DNA / 2′。通过诱导或保留该特定基因的突变而能够诱导或抑制涉及眼功能的特定基因表达的OMeRNA寡核苷酸,及其用作预防或治疗眼部疾病的治疗组合物的用途。

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