Carrier system for specific artery wall gene delivery
    1.
    发明授权
    Carrier system for specific artery wall gene delivery 有权
    特异性动脉壁基因递送载体系统

    公开(公告)号:US07264969B1

    公开(公告)日:2007-09-04

    申请号:US10416381

    申请日:2001-11-09

    CPC classification number: A61K48/0075

    Abstract: An artery wall binding peptide (AWBP) based on the artery wall cell-binding domain of apolipoprotein B-100 was conjugated to a cationic backbone configured for forming a complex with a nucleic acid to produce a composition that enhances gene transfer to artery wall cells. An illustrative cationic backbone is poly(ethylene glycol)-grafted-poly(L-lysine) (PEG-g-PLL). Methods of making and using the composition for gene transfer are also described.

    Abstract translation: 基于载脂蛋白B-100的动脉壁细胞结合结构域的动脉壁结合肽(AWBP)缀合到阳离子骨架上,其配置用于与核酸形成复合物,以产生增强基因转移到动脉壁细胞的组合物。 一个说明性的阳离子骨架是聚(乙二醇)移植的聚(L-赖氨酸)(PEG-g-PLL)。 还描述了制备和使用用于基因转移的组合物的方法。

    Hypoxia inducible VEGF plasmid for ischemic disease
    5.
    发明授权
    Hypoxia inducible VEGF plasmid for ischemic disease 失效
    缺氧诱导型VEGF质粒缺血性疾病

    公开(公告)号:US07638322B2

    公开(公告)日:2009-12-29

    申请号:US10546115

    申请日:2004-02-23

    CPC classification number: C07K14/52 A61K48/0058 C12N15/85 C12N2830/002

    Abstract: Plasmids useful for treating ischemic disease, such as ischemic heart disease, are described. The plasmids express vascular endothelial growth factor (VEGF) under the control of a promoter (RTP801) that is up-regulated under hypoxic conditions. Pharmaceutical compositions for treating ischemic disease include mixtures of the hypoxia-regulated VEGF plasmids and pharmaceutically acceptable carriers. Methods for treating ischemic disease include administering such pharmaceutical compositions to a person in need of such treatment.

    Abstract translation: 描述了可用于治疗缺血性疾病如缺血性心脏病的质粒。 质粒表达在缺氧条件下上调的启动子(RTP801)控制下的血管内皮生长因子(VEGF)。 用于治疗缺血性疾病的药物组合物包括缺氧调节的VEGF质粒和药学上可接受的载体的混合物。 治疗缺血性疾病的方法包括将这种药物组合物给予需要这种治疗的人。

    Linear polyethylenimine-sterol conjugates for gene delivery

    公开(公告)号:US07183263B2

    公开(公告)日:2007-02-27

    申请号:US10623020

    申请日:2003-07-17

    CPC classification number: C07J9/00 A61K47/59 A61K48/0041

    Abstract: Linear polyethylenimine was modified with sterols, such as cholesterol, in three different geometries: linear shaped (L), T-shaped (T), and a combined linear- and T-shaped (LT), to result in linear polyethylenimine-sterol conjugates. These conjugates were mixed with nucleic acids to form complexes for delivery of the nucleic acids into cells. Mammalian cells transfected with these complexes showed protein expression levels higher than linear polyethylenimine alone, and twice that of branched polyethylenimine, but without any significant loss in cell viability. Methods of making these compositions and methods of using them for gene delivery are also described.

    CLEAVABLE MODIFICATIONS TO REDUCIBLE POLY (AMIDO ETHYLENIMINES)S TO ENHANCE NUCLEOTIDE DELIVERY
    7.
    发明申请
    CLEAVABLE MODIFICATIONS TO REDUCIBLE POLY (AMIDO ETHYLENIMINES)S TO ENHANCE NUCLEOTIDE DELIVERY 审中-公开
    可减少聚氨酯(AMIDO乙烯)的改进可以提高核子递送

    公开(公告)号:US20130149783A1

    公开(公告)日:2013-06-13

    申请号:US13635479

    申请日:2011-03-16

    Abstract: Polyplex formulations were prepared using p(TETA/CBA), its PEGylated analog, p(TETA/CBA)-g-PEG2k, and mixtures of the two species at 10/90 and 50/50 wt %, respectively. Increasing PEG wt % inhibited polyplex formation. This work demonstrates the feasibility of preparing homogenous polyplexes by altering the PEG wt % using a mixture of p(TETA/CBA) and p(TETA/CBA)-g-PEG2k products. Further, a single-step method of making p(TETA/CBA)-g-PEG2k is disclosed.

    Abstract translation: 使用p(TETA / CBA),其聚乙二醇化类似物,p(TETA / CBA)-g-PEG2k以及两种物质的混合物分别在10/90和50/50重量%下分别制备多功能配方。 增加PEG wt%抑制聚合物形成。 该工作证明了通过使用p(TETA / CBA)和p(TETA / CBA)-g-PEG2k产物的混合物改变PEG wt%来制备均匀复合物的可行性。 此外,公开了制备p(TETA / CBA)-g-PEG2k的单步方法。

    Arginine-conjugated bioreducible poly(disulfide amine) polymers for gene delivery systems
    8.
    发明授权
    Arginine-conjugated bioreducible poly(disulfide amine) polymers for gene delivery systems 有权
    用于基因递送系统的精氨酸缀合的生物还原性聚(二硫化物胺)聚合物

    公开(公告)号:US08153154B2

    公开(公告)日:2012-04-10

    申请号:US12370515

    申请日:2009-02-12

    Abstract: An arginine-grafted bioreducible poly(disulfide amine) (“ABP”) as a reagent for efficient and nontoxic gene delivery is described. ABP forms positively charged nano-particles of less than 200 nm with plasmid DNA. ABP is biodegraded under reducing conditions, such as the cytoplasm. ABP exhibits much higher transfection efficiency than polyethyleneimine in mammalian cells and exhibits no cytotoxicity.

    Abstract translation: 描述了精氨酸接枝的可生物还原的聚(二硫化胺)(“ABP”)作为有效和无毒的基因递送的试剂。 ABP与质粒DNA形成小于200nm的带正电荷的纳米颗粒。 ABP在还原条件下生物降解,如细胞质。 与哺乳动物细胞中的聚乙烯亚胺相比,ABP的转染效率高得多,不显示细胞毒性。

    Hypoxia inducible VEGF plasmid for ischemic disease
    9.
    发明授权
    Hypoxia inducible VEGF plasmid for ischemic disease 有权
    缺氧诱导型VEGF质粒缺血性疾病

    公开(公告)号:US07998941B2

    公开(公告)日:2011-08-16

    申请号:US12649141

    申请日:2009-12-29

    CPC classification number: C07K14/52 A61K48/0058 C12N15/85 C12N2830/002

    Abstract: Plasmids useful for treating ischemic disease, such as ischemic heart disease, are described. The plasmids express vascular endothelial growth factor (VEGF) under the control of a promoter (RTP801) that is up-regulated under hypoxic conditions. Pharmaceutical compositions for treating ischemic disease include mixtures of the hypoxia-regulated VEGF plasmids and pharmaceutically acceptable carriers. Methods for treating ischemic disease include administering such pharmaceutical compositions to a person in need of such treatment.

    Abstract translation: 描述了可用于治疗缺血性疾病如缺血性心脏病的质粒。 质粒在缺氧条件下上调的启动子(RTP801)的控制下表达血管内皮生长因子(VEGF)。 用于治疗缺血性疾病的药物组合物包括缺氧调节的VEGF质粒和药学上可接受的载体的混合物。 用于治疗缺血性疾病的方法包括将这种药物组合物给予需要这种治疗的人。

    Linear polyethylenimine-sterol conjugates for gene delivery
    10.
    发明授权
    Linear polyethylenimine-sterol conjugates for gene delivery 有权
    用于基因递送的线性聚乙烯亚胺 - 甾醇缀合物

    公开(公告)号:US07863470B2

    公开(公告)日:2011-01-04

    申请号:US11711933

    申请日:2007-02-27

    CPC classification number: C07J9/00 A61K47/59 A61K48/0041

    Abstract: Linear polyethylenimine was modified with sterols, such as cholesterol, in three different geometries: linear shaped (L), T-shaped (T), and a combined linear- and T-shaped (LT), to result in linear polyethylenimine-sterol conjugates. These conjugates were mixed with nucleic acids to form complexes for delivery of the nucleic acids into cells. Mammalian cells transfected with these complexes showed protein expression levels higher than linear polyethylenimine alone, and twice that of branched polyethylenimine, but without any significant loss in cell viability. Methods of making these compositions and methods of using them for gene delivery are also described.

    Abstract translation: 线性聚乙烯亚胺用甾醇如胆固醇在三种不同的几何形状中进行了改性:线形(L),T形(T)和组合的线形和T形(LT),得到线性聚乙烯亚胺 - 固醇共轭物 。 将这些缀合物与核酸混合以形成用于将核酸递送到细胞中的复合物。 用这些复合物转染的哺乳动物细胞显示蛋白质表达水平高于单独的线性聚乙烯亚胺,是支链聚乙烯亚胺的两倍,但细胞活力没有任何显着的损失。 还描述了制备这些组合物的方法和使用它们进行基因递送的方法。

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