Functional synthetic molecules and macromolecules for gene delivery
    1.
    发明授权
    Functional synthetic molecules and macromolecules for gene delivery 失效
    用于基因递送的功能性合成分子和大分子

    公开(公告)号:US07507859B2

    公开(公告)日:2009-03-24

    申请号:US10559349

    申请日:2004-06-16

    摘要: The present invention describes a synthetic non-viral vector composition for gene therapy and the use of such compositions for in vitro, ex vivo and/or in vivo transfer of genetic material. The invention proposes a pharmaceutical composition containing 1) a non-cationic amphiphilic molecule or macromolecule and its use for delivery of nucleic acids or 2) a cationic amphiphilic molecule or macromolecule that transforms from a cationic entity to an anionic, neutral, or zwitterionic entity by a chemical, photochemical, or biological reaction and its use for delivery of nucleic acids. Moreover this invention describes the use of these non-viral vector compositions in conjunction with a surface to mediate the delivery of nucleic acids. An additional embodiment is the formation of a hydrogel with these compositions and the use of this hydrogel for the delivery of genetic material. A further embodiment of this invention is the use of a change in ionic strength for the delivery of genetic material.

    摘要翻译: 本发明描述了用于基因治疗的合成非病毒载体组合物和用于遗传物质的体外,离体和/或体内转移的这些组合物。 本发明提出一种药物组合物,其含有1)非阳离子两亲性分子或大分子及其用于递送核酸的用途,或2)阳离子两亲分子或大分子,其通过以下方式从阳离子实体转变为阴离子,中性或两性离子的实体: 化学,光化学或生物反应及其用于递送核酸的用途。 此外,本发明描述了这些非病毒载体组合物与表面结合以介导核酸递送的用途。 另外的实施方案是用这些组合物形成水凝胶,以及使用该水凝胶来输送遗传物质。 本发明的另一个实施方案是使用离子强度的变化来传递遗传物质。

    Crosslinked gels comprising polyalkyleneimines, and their uses as medical devices

    公开(公告)号:US09393344B2

    公开(公告)日:2016-07-19

    申请号:US11653433

    申请日:2007-01-11

    摘要: One aspect of the present invention generally relates to methods of sealing a wound or tissue plane or filling a void splace. In a preferred embodiment, the wound is an ophthalmic, pleural or dural wound. In certain instances, the compositions used to seal the wound or tissue plane comprises a polyalkyleneimine. In a preferred embodiment, the polyalkyleneimine is polyethyleneimine. Treatment of the polyethyleneimine with a cross-linking reagent causes the polyethyleneimine polymers to polymerize forming a seal. In certain instances, the cross-linking reagent is a polyethylene glycol having reactive terminal groups. In certain instances, the reactive terminal groups are activated esters, such as N-hydroxy succinimide ester. In certain instances, the reactive terminal groups are isocyanates. In certain instances, the polyethyleneimine has a lysine, cysteine, isocysteine or other nucleophilic group attached to the periphery of the polymer. In certain instances, the polyethyleneimine is mixed with a second polymer, such as a polyethylene glycol containing nucleophilic groups. In certain instances, the compositions used to seal the wound or tissue plane are formed by reacting a polyalkyleneimine bearing electrophilic groups with a cross-linking reagent containing nucleophilic groups. In certain instances, the electrophilic groups on the polyalkyleneimine are activated esters, such as N-hydroxy succinimide ester. In certain instances, the compositions used to seal the wound or tissue plane are formed by reacting a polyalkyleneimine bearing photopolymerizable groups with ultraviolet or visibile light. Compositions used to seal the wound which contain PEI or a derivative of PEI are found to adhere tightly to the tissue. Other aspects of the present invention relate to methods of filling a void of a patient or adhering tissue. In certain instances, the methods use a polyalkyleneimine. In a preferred embodiment, the polyalkyleneimine is polyethyleneimine. Another aspect of the present invention relates to a polymeric composition formed by exposing a polyalkyleneimine to an activated polyalkylene glycol. In certain instances, the composition is attached to mammalian tissue.