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89 条记录 (耗时 0.026 秒)

    Cationic amphiphiles containing spermine or spermidine cationic group for intracellular delivery of therapeutic molecules
    81.
    发明授权
    Cationic amphiphiles containing spermine or spermidine cationic group for intracellular delivery of therapeutic molecules 失效
    含有精胺或亚精胺阳离子基团的阳离子两亲物用于细胞内递送治疗分子

    公开(公告)号：US5783565A

    公开(公告)日：1998-07-21

    申请号：US595375

    申请日：1996-02-01

    申请人： Edward R. Lee ,  David J. Harris ,  Craig S. Siegel ,  Seng H. Cheng ,  Simon J. Eastman ,  John Marshall ,  Ronald K. Scheule

    发明人： Edward R. Lee ,  David J. Harris ,  Craig S. Siegel ,  Seng H. Cheng ,  Simon J. Eastman ,  John Marshall ,  Ronald K. Scheule

    IPC分类号： A61K9/127 ,  A61K47/48 ,  A61K48/00 ,  C07J41/00 ,  C07J43/00 ,  C12N15/88 ,  B01F17/16 ,  C12N15/00

    CPC分类号： B82Y5/00 ,  A61K47/48023 ,  A61K47/48038 ,  A61K47/48046 ,  A61K47/48123 ,  A61K48/00 ,  A61K9/1272 ,  C07J41/0005 ,  C07J41/0055 ,  C07J41/0061 ,  C07J43/003 ,  C12N15/88 ,  Y10S516/07

    摘要： Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from alkyl or acyl groups; and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile. Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation. Additionally, targeting of organs for gene therapy by intravenous administration of therapeutic compositions is described.

    摘要翻译： 提供了新的阳离子两亲物，其促进生物活性（治疗性）分子进入细胞。 两亲物含有衍生自类固醇的单亲或二烷基胺或烷基或酰基的亲脂基团; 和在生理pH下可质子化的阳离子基团，衍生自胺，烷基胺或多烷基胺。 还提供了通常通过使一种或多种阳离子两亲物的分散体与治疗分子接触而制备的治疗组合物。 根据本发明的实践可以递送到细胞中的治疗分子包括DNA，RNA和多肽。 本发明治疗组合物的代表性用途包括提供基因治疗以及向细胞递送反义多核苷酸或生物活性多肽。 关于基因治疗的治疗组合物，通常以与阳离子两亲物络合的质粒的形式提供DNA。 还公开了新型和高效的质粒构建体，包括在为炎症复杂的临床病症提供基因治疗方面特别有效的质粒构建体。 另外，描述了通过静脉内施用治疗组合物靶向用于基因治疗的器官。

    Cholesterol conjugates of 2'5'-oligoadenylate derivatives and antiviral uses thereof
    85.
    发明授权
    Cholesterol conjugates of 2'5'-oligoadenylate derivatives and antiviral uses thereof 失效
    2'5'-寡腺苷酸衍生物的胆固醇结合物及其抗病毒用途

    公开(公告)号：US5643889A

    公开(公告)日：1997-07-01

    申请号：US306274

    申请日：1994-09-14

    申请人： Robert J. Suhadolnik ,  Wolfgang Pfleiderer

    发明人： Robert J. Suhadolnik ,  Wolfgang Pfleiderer

    IPC分类号： A61K47/48 ,  C07H21/00 ,  C07H23/00 ,  C07J43/00 ,  C07J51/00 ,  A01G7/06 ,  A61K31/70

    CPC分类号： A61K31/70 ,  A61K31/7115 ,  A61K31/712 ,  A61K47/48023 ,  A61K47/48092 ,  A61K47/48107 ,  A61K47/48123 ,  A61K47/48146 ,  C07H21/00 ,  C07H23/00 ,  C07J43/003 ,  C07J51/00

    摘要： A cholesterol-cordycepin conjugate having the formula ##STR1## wherein: n is an integer from 1 to 8; R.sub.1 is selected from the group of consisting of T, T' and Y; T is ##STR2## T' is ##STR3## where x is an integer from 1 to 18; Y is ##STR4## where m is zero, 1, 2, or 3; each R.sub.2 is independently selected from the group consisting of oxygen and sulfur; each R.sub.3 is independently selected from the group consisting of hydrogen and hydroxyl; R.sub.4 is selected from the group consisting of hydrogen, hydroxyl and T or T'; R.sub.5 is selected from the group consisting of hydrogen, hydroxyl and T or T'; provided that all R.sub.1, R.sub.4 and R.sub.5 may not be T or T'; at least one R.sub.3 is hydrogen or R.sub.4 is hydrogen; and at least one of R.sub.1, R.sub.4 and R.sub.5 must be T or T'; or a water soluble salt thereof. The compounds possess increased antiviral activity and/or metabolic stability.

    摘要翻译： 具有下式的胆固醇 - 虫草素缀合物其中：n是1至8的整数; R1选自T，T'和Y组成的组; T是 T'是，其中x是从1到18的整数; Y是，其中m是0,1,2或3; 每个R 2独立地选自氧和硫; 每个R 3独立地选自氢和羟基; R4选自氢，羟基和T或T'; R5选自氢，羟基和T或T'; 条件是所有R1，R4和R5可能不是T或T'; 至少一个R 3是氢或R 4是氢; 并且R1，R4和R5中的至少一个必须是T或T'; 或其水溶性盐。 该化合物具有增加的抗病毒活性和/或代谢稳定性。

    Steroid modified oligonucleotides
    87.
    发明授权
    Steroid modified oligonucleotides 失效
    类固醇修饰寡核苷酸

    公开(公告)号：US5416203A

    公开(公告)日：1995-05-16

    申请号：US97320

    申请日：1993-07-26

    申请人： Robert L. Letsinger

    发明人： Robert L. Letsinger

    IPC分类号： A61K31/704 ,  A61K31/7088 ,  A61P31/12 ,  A61P31/18 ,  C07H21/00 ,  C07J43/00 ,  C07J51/00

    CPC分类号： C07H21/00 ,  A61K47/48038 ,  A61K47/48123 ,  C07J51/00

    摘要： An oligonucleotide conjugated to a steroid the oligonucleotide comprising: ##STR1## wherein A is selected from the group consisting of an aliphatic alkyl, branched aliphatic alkyl and an alkyl (branched) chain of 2 to 18 carbon atoms, R is selected from the group consisting of H and lower alkyl up to 12 carbon atoms; B is a naturally occurring base, the steroid is bound to the oligonucleotide utilizing the naturally occurring bases through an (-oxycarbonyl-amino)-decylamine-moiety, and NUC is an oligonucleotide.

    摘要翻译： 与寡核苷酸缀合的寡核苷酸包含：其中A选自脂族烷基，支链脂族烷基和2至18个碳原子的烷基（支链），R选自： 的H和低至12个碳原子的低级烷基; B是天然存在的碱基，使用天然存在的碱通过（ - 羰基 - 氨基） - 十九胺 - 部分结合寡核苷酸，并且NUC是寡核苷酸。