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    TARGETED DELIVERY OF GENES ENCODING SECRETORY PROTEINS
    13.
    发明申请
    TARGETED DELIVERY OF GENES ENCODING SECRETORY PROTEINS 审中-公开
    目标交付编码秘密蛋白的基因

    公开(公告)号：WO1992022635A1

    公开(公告)日：1992-12-23

    申请号：PCT/US1992004565

    申请日：1992-06-05

    Applicant: UNIVERSITY OF CONNECTICUT ,  THE BOARD OF REGENTS acting for and on behalf of ...

    Inventor： UNIVERSITY OF CONNECTICUT ,  THE BOARD OF REGENTS acting for and on behalf of ... ,  WU, George, Y. ,  WILSON, James, M. ,  WU, Catherine, H.

    IPC: A61K47/48

    CPC classification number: A61K48/00 ,  A61K47/645 ,  C12N15/87

    Abstract: Molecular complexes for targeting a gene encoding a secretory protein to a specific cell in vivo and obtaining secretion of the protein by the targeted cell are disclosed. An expressible gene encoding a desired secretory protein is complexed to a conjugate of a cell-specific binding agent and a gene-binding agent. The cell-specific binding agent is specific for a cellular surface structure which mediates internalization of ligands by endocytosis. An example is the asialoglycoprotein receptor of hepatocytes. The gene-binding agent is a compound such as a polycation which stably complexes the gene under extracellular conditions and releases the gene under intracellular conditions so that it can function within a cell. The molecular complex is stable and soluble in physiological fluids and can be used in gene therapy to selectively transfect cells in vivo to provide for production and secretion of a desired secretory protein.

    Abstract translation: 公开了用于将编码分泌蛋白的基因靶向体内特定细胞并获得目标细胞分泌蛋白质的分子复合物。 编码所需分泌蛋白的可表达基因与细胞特异性结合剂和基因结合剂的缀合物复合。 细胞特异性结合剂对通过内吞作用介导配体内化的细胞表面结构是特异性的。 一个例子是肝细胞的脱唾液酸糖蛋白受体。 基因结合剂是诸如聚阳离子的化合物，其在细胞外条件下稳定地复合基因并在细胞内条件下释放基因，使得其可以在细胞内起作用。 分子复合物稳定并且可溶于生理液体，可用于基因治疗以选择性转染体内细胞以提供所需分泌蛋白的产生和分泌。

    SURFACE-MODIFIED NANOPARTICLES AND METHOD OF MAKING AND USING SAME
    14.
    发明申请
    SURFACE-MODIFIED NANOPARTICLES AND METHOD OF MAKING AND USING SAME 审中-公开
    表面改性纳米粒子及其制备方法和使用方法

    公开(公告)号：WO1996020698A2

    公开(公告)日：1996-07-11

    申请号：PCT/US1996000476

    申请日：1996-01-04

    Applicant: THE BOARD OF REGENTS acting for and on behalf of THE UNIVERSITY OF MICHIGAN ,  LEVY, Robert, J. ,  LABHASETWAR, Vinod, D. ,  SONG, Cunxian, S.

    Inventor： THE BOARD OF REGENTS acting for and on behalf of THE UNIVERSITY OF MICHIGAN

    IPC: A61K09/51

    CPC classification number: A61K9/5153

    Abstract: Biodegradable controlled release nanoparticles as sustained release bioactive agent delivery vehicles include surface modifying agents to target binding of the nanoparticles to tissues or cells of living systems, to enhance nanoparticle sustained release properties, and to protect nanoparticle-incorporated bioactive agents. Unique methods of making small (10 nm to 15 nm, and preferably 20 nm to 35 nm) nanoparticles having a narrow size distribution which can be surface-modified after the nanoparticles are formed is described. Techniques for modifying the surface include a lyophilization technique to produce a physically adsorbed coating and epoxy-derivatization to functionalize the surface of the nanoparticles to covalently bind molecules of interest. The manoparticles may also comprise hydroxy-terminated or epoxide-terminated and/or activated multiblock copolymers, having hydrophobic segments which may be polycaprolactone and hydrophilic segments. The nanoparticles are useful for local intravascular administration of smooth muscle inhibitors and antithrombogenic agents as part of interventional cardiac or vascular catheterization such as a balloon angioplasty procedure; direct application to tissues and/or cells for gene therapy, such as the delivery of osteotropic genes or gene segments into bone progenitor cells; or oral administration in an enteric capsule for delivery of protein/peptide based vaccines.

    Abstract translation: 作为持续释放的生物活性剂递送载体的可生物降解的控制释放纳米颗粒包括靶向纳米颗粒与生物体系的组织或细胞的结合的表面改性剂，以增强纳米颗粒持续释放性质，并保护纳米颗粒引入的生物活性剂。 描述了在形成纳米颗粒之后可以进行表面改性的具有窄尺寸分布的小（10nm至15nm，优选20nm至35nm）纳米颗粒的独特方法。 用于改性表面的技术包括冷冻干燥技术以产生物理吸附的涂层和环氧衍生化，以官能化纳米颗粒的表面以共价结合感兴趣的分子。 人造粒体还可包含羟基封端或环氧化物封端和/或活化的多嵌段共聚物，其具有可以是聚己内酯和亲水链段的疏水链段。 纳米颗粒可用于局部血管内施用平滑肌抑制剂和抗血栓形成剂，作为介入性心脏或血管导管插入术的一部分，例如球囊血管成形术程序; 直接应用于用于基因治疗的组织和/或细胞，例如将骨质基因或基因片段递送到骨祖细胞中; 或用于输送蛋白质/肽基疫苗的肠溶胶囊中的口服给药。

    Cystic fibrosis gene
    19.
    发明公开
    Cystic fibrosis gene 失效
    Zystische-Fibrose Gen

    公开(公告)号：EP1418183A1

    公开(公告)日：2004-05-12

    申请号：EP03078489.6

    申请日：1990-08-20

    Applicant: HSC Research Development Corporation ,  THE BOARD OF REGENTS ACTING FOR AND ON BEHALF OF THE UNIVERSITY OF MICHIGAN

    Inventor： Tsui, Lap-Chee ,  Riordan, John R. ,  Collins, Francis S. ,  Rommens, Johanna M. ,  Iannuzzi, Michael C. ,  Kerem, Bat-Sheva ,  Drumm, Mitchell L. ,  Buchwald, Manual

    IPC: C07K14/47 ,  C12N15/12 ,  C12Q1/68

    CPC classification number: C07K16/28 ,  A01K2217/05 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K48/00 ,  B82Y5/00 ,  C07K14/4712 ,  C07K16/18 ,  C12N15/8509

    Abstract: A purified RNA molecule comprises an RNA sequence corresponding to a DNA molecule comprising an intronless DNA sequence selected from:

    (a) DNA sequences which correspond to the DNA sequence of Figure 1 from amino acid residue position 1 to position 1480;
    (b) DNA sequences encoding normal CFTR polypeptide having the sequence according to Figure 1 for amino acid residue positions from 1 to 1480;
    (c) DNA sequences which correspond to a fragment of the sequence of Figure 1 including at least 16 sequential nucleotides between amino acid residue positions 1 and 1480;
    (d) DNA sequences which comprise at least 16 nucleotides and encode a fragment of the amino acid sequence of Figure 1; and
    (e) DNA sequences encoding an epitope encoded by at least 18 sequential nucleotides in the sequence of Figure 1 between amino acid residue positions I and 1480.

    Similar RNA molecules are also disclosed, as are nucleic acid probes and polypeptides based on the RNA sequences.
    A method of screening for cystic fibrosis includes use of the nucleotide probes described.

    Abstract translation: 纯化的RNA分子包含对应于包含不含内含DNA序列的DNA分子的RNA序列，所述内含子DNA序列选自：（a）对应于图1的DNA序列的DNA序列，其氨基酸残基位置1至位置1480; （b）编码具有根据图1的序列的1至1480氨基酸残基的正常CFTR多肽的DNA序列; （c）对应于图1的序列的片段的DNA序列，其包含氨基酸残基1至1480之间的至少16个连续核苷酸; （d）包含至少16个核苷酸并编码图1的氨基酸序列的片段的DNA序列; 和（e）编码由图1的序列中的至少18个连续核苷酸编码的表位的氨基酸残基位置I和1480之间的DNA序列。同样公开了类似的RNA分子，核酸探针和多肽基 对RNA序列。 筛选囊性纤维化的方法包括使用所述的核苷酸探针。

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