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    Cationic amphiphiles for intracellular delivery of therapeutic molecules
    3.
    发明授权
    Cationic amphiphiles for intracellular delivery of therapeutic molecules 有权
    阳离子两亲物用于细胞内递送治疗分子

    公开(公告)号：US06383814B1

    公开(公告)日：2002-05-07

    申请号：US09228232

    申请日：1999-01-11

    申请人： Edward R. Lee ,  David J. Harris ,  Craig S. Siegel ,  Mathieu B. Lane ,  Shirley C. Hubbard ,  Seng H. Cheng ,  Simon J. Eastman ,  John Marshall ,  Ronald K Scheule ,  Nelson S. Yew

    发明人： Edward R. Lee ,  David J. Harris ,  Craig S. Siegel ,  Mathieu B. Lane ,  Shirley C. Hubbard ,  Seng H. Cheng ,  Simon J. Eastman ,  John Marshall ,  Ronald K Scheule ,  Nelson S. Yew

    IPC分类号： C12N1588

    CPC分类号： C12N15/88 ,  A61K9/1272 ,  A61K47/542 ,  A61K47/543 ,  A61K47/554 ,  A61K48/00 ,  C07J41/0005 ,  C07J41/0055 ,  C07J41/0061 ,  C07J43/003

    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。 关于用于基因治疗的治疗组合物，DNA通常以用于与阳离子两亲物络合的质粒的形式提供。还公开了新的和高效的质粒构建体，包括在为临床条件提供基因治疗方面特别有效的 炎症复杂。 此外，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。

    Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
    4.
    发明授权
    Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules 失效
    阳离子两亲物和质粒用于细胞内递送治疗分子

    公开(公告)号：US5910487A

    公开(公告)日：1999-06-08

    申请号：US546205

    申请日：1995-10-20

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

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

    IPC分类号： A61K9/127 ,  A61K47/48 ,  A61K48/00 ,  C07J41/00 ,  C07J43/00 ,  C12N15/88 ,  A61K37/00

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

    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。

    Transgene expression system for increased persistence
    9.
    发明授权
    Transgene expression system for increased persistence 失效
    转基因表达系统增加持久性

    公开(公告)号：US5981275A

    公开(公告)日：1999-11-09

    申请号：US839552

    申请日：1997-04-14

    申请人： Donna Armentano ,  John Marshall ,  Nelson S. Yew ,  Seng H. Cheng ,  Richard J. Gregory

    发明人： Donna Armentano ,  John Marshall ,  Nelson S. Yew ,  Seng H. Cheng ,  Richard J. Gregory

    IPC分类号： C12N15/09 ,  A61K35/76 ,  A61K48/00 ,  A61P11/00 ,  C07K14/47 ,  C12N1/15 ,  C12N1/19 ,  C12N1/21 ,  C12N5/10 ,  C12N15/861 ,  C12N15/00

    CPC分类号： C12N15/86 ,  C07K14/4712 ,  A61K48/00 ,  C12N2710/10343 ,  C12N2830/00 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/60 ,  C12N2830/85

    摘要： The invention is directed to a transgene expression system comprising a transcription unit which contains a transgene operably linked to expression control sequences, preferably the CMV promoter, and which is delivered simultaneously with all or part of the adenovirus E4 genomic region to a cell in order to facilitate persistent expression of the transgene. The components of the transgene expression system can be delivered by vectors including plasmids and/or viruses and may be complexed with cationic amphiphiles to facilitate entry into a cell. The invention is also directed to methods for the production of the transgene expression system. The invention is further directed to compositions that contain the transgene expression system and to methods for the use of such compositions to deliver transgenes encoding biologically active proteins to cells.

    摘要翻译： 本发明涉及包含转录单元的转基因表达系统，该转录单元含有可操作地连接到表达调控序列，优选CMV启动子的转基因，并且其与全部或部分腺病毒E4基因组区域同时递送至细胞，以便 促进转基因的持续表达。 转基因表达系统的组分可以通过包括质粒和/或病毒的载体递送，并且可以与阳离子两亲物复合以便于进入细胞。 本发明还涉及生产转基因表达系统的方法。 本发明还涉及含有转基因表达系统的组合物以及使用这种组合物将编码生物活性蛋白质的转基因递送至细胞的方法。