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1. 发明授权

US07279326B2 Composition for delivery of a mitochondrial genome to a cell 有权
标题翻译：用于将线粒体基因组递送至细胞的组合物
公开(公告)号：US07279326B2
公开(公告)日：2007-10-09
申请号：US10485407
申请日：2002-07-31
申请人： Volkmar Weissig , Vladimir Torchilin
发明人： Volkmar Weissig , Vladimir Torchilin
IPC分类号： C12N15/00
CPC分类号： A61K48/00 , C12N15/87
摘要： The invention provides composition and methods for delivering a composition according to the invention comprising a wild-type (wt) mitochondrial DNA (mtDNA) molecule to a mammalian cell. The wt-mtDNA molecule is a functional mtDNA molecule that includes the entire mitochondrial genome. The wt-mtDNA molecule in the composition of the invention has a mitochondrial leader sequence (MLS) peptide attached to facilitate the uptake of the mtDNA molecule into a mitochondrion of the mammalian cell. The delivery of the wt-mtDNA/MLS peptide complex is used as a general replenishment therapy for conditions attributed to mtDNA defects, independent of the specific defect in the mtDNA.
摘要翻译：本发明提供了将包含野生型（wt）线粒体DNA（mtDNA）分子的根据本发明的组合物递送至哺乳动物细胞的组合物和方法。 wt-mtDNA分子是包括整个线粒体基因组的功能性mtDNA分子。本发明组合物中的wt-mtDNA分子具有连接的线粒体前导序列（MLS）肽，以促进mtDNA分子摄取到哺乳动物细胞的线粒体中。将wt-mtDNA / MLS肽复合物的递送用作归因于mtDNA缺陷的条件的一般补充疗法，与mtDNA中的特异性缺陷无关。

2. 发明申请

US20100260676A1 PRECISION-GUIDED NANOPARTICLE SYSTEMS FOR DRUG DELIVERY 有权
标题翻译：用于药物递送的精确指导的纳米系统
公开(公告)号：US20100260676A1
公开(公告)日：2010-10-14
申请号：US12526297
申请日：2008-02-11
申请人： Robert N. Hanson , Mansoor Amiji , Volkmar Weissig
发明人： Robert N. Hanson , Mansoor Amiji , Volkmar Weissig
IPC分类号： A61K49/00 , A61K9/14 , A61K39/395 , A61K31/28 , A61K31/295 , A61K31/565 , A61K31/198 , A61K38/19 , A61K31/195
CPC分类号： A61K49/0002 , A61K9/51 , A61K47/6923 , A61K47/6929 , A61K51/1244 , B82Y5/00
摘要： A method of preparing multifunctionalized nanoparticles involves using a modular system of half-linkers to attach functional moieties that serve to deliver the nanoparticles to a desired target, exert an effect at the target, or track the nanoparticles within a cell or an animal. The modular chemistry of the half-linker system permits the custom design and synthesis of functionalized nanoparticles bearing multiple groups and therefore results in precise delivery to desired cell types and intracellular locations. The functionalized nanoparticles can be used to treat or diagnose a variety of medical conditions, including neoplastic diseases, infectious diseases, and chronic diseases.
摘要翻译：制备多功能纳米颗粒的方法包括使用半连接体的模块化系统来附着功能部分，其用于将纳米颗粒递送到期望的靶，在靶上发挥作用，或跟踪细胞或动物内的纳米颗粒。半连接器系统的模块化化学允许定制设计和合成具有多个组的官能化纳米颗粒，因此导致精确递送到期望的细胞类型和细胞内位置。功能化纳米颗粒可用于治疗或诊断各种医疗状况，包括肿瘤疾病，传染病和慢性疾病。

3. 发明申请

US20080095834A1 Mitochondriotropic Phospholipid Vesicles 审中-公开
标题翻译：线粒体磷脂囊泡
公开(公告)号：US20080095834A1
公开(公告)日：2008-04-24
申请号：US11885419
申请日：2006-03-02
申请人： Volkmar Weissig , Sarathi Boddapati , Robert Hanson , Vladimir Torchilin
发明人： Volkmar Weissig , Sarathi Boddapati , Robert Hanson , Vladimir Torchilin
IPC分类号： A61K9/127 , A61K31/70 , A61P43/00 , A61K38/00
CPC分类号： A61K9/1272
摘要： Mitochondriotropic phospholipid vesicles, i.e., mitochondriotropic liposomes, that comprise a hydrophobized amphiphilic delocalized cation, such as those comprising, e.g., a triphenylphosphonium or a quinolinium moiety, incorporated into the phospholipid membrane of the vesicles, or liposomes, are disclosed. The hydrophobized portion of the amphiphilic delocalized cation, e.g., a fatty acid or other phospholipid derivative, is embedded in the phospholipid membrane of the liposome, and the amphiphilic portion of the cation is exposed on the surface of the liposome. Mitochondriotropic liposomes constitute a mitochondria-targeted drug delivery system, permitting the transport of a high payload of therapeutic water-soluble molecules in their native (i.e., active) state specifically and exclusively to mitochondria in living mammalian cells.
摘要翻译：公开了包含疏水化两亲离域阳离子（例如掺入到囊泡的磷脂膜中的三苯基鏻或喹啉鎓部分的那些）的线粒体磷脂囊泡，即线粒体二次性脂质体，或脂质体。两亲离域阳离子的疏水化部分，例如脂肪酸或其它磷脂衍生物，被包埋在脂质体的磷脂膜中，阳离子的两亲部分暴露在脂质体的表面上。线粒体脂质体构成线粒体靶向药物递送系统，允许将其天然（即活性）状态中的治疗性水溶性分子的高有效载体特异性地转移至活的哺乳动物细胞中的线粒体。

4. 发明授权

US09056129B2 Precision-guided nanoparticle systems for drug delivery 有权
标题翻译：用于药物递送的精确导向纳米颗粒系统
公开(公告)号：US09056129B2
公开(公告)日：2015-06-16
申请号：US12526297
申请日：2008-02-11
申请人： Robert N. Hanson , Mansoor Amiji , Volkmar Weissig
发明人： Robert N. Hanson , Mansoor Amiji , Volkmar Weissig
IPC分类号： A61K9/14 , A61K49/00 , A61K9/51 , A61K47/48 , A61K51/12 , B82Y5/00
CPC分类号： A61K49/0002 , A61K9/51 , A61K47/6923 , A61K47/6929 , A61K51/1244 , B82Y5/00
摘要： A method of preparing multifunctionalized nanoparticles involves using a modular system of half-linkers to attach functional moieties that serve to deliver the nanoparticles to a desired target, exert an effect at the target, or track the nanoparticles within a cell or an animal. The modular chemistry of the half-linker system permits the custom design and synthesis of functionalized nanoparticles bearing multiple groups and therefore results in precise delivery to desired cell types and intracellular locations. The functionalized nanoparticles can be used to treat or diagnose a variety of medical conditions, including neoplastic diseases, infectious diseases, and chronic diseases.
摘要翻译：制备多功能纳米颗粒的方法包括使用半连接体的模块化系统来附着功能部分，其用于将纳米颗粒递送到期望的靶，在靶上发挥作用，或跟踪细胞或动物内的纳米颗粒。半连接器系统的模块化化学允许定制设计和合成具有多个组的官能化纳米颗粒，因此导致精确递送到期望的细胞类型和细胞内位置。功能化纳米颗粒可用于治疗或诊断各种医疗状况，包括肿瘤疾病，传染病和慢性疾病。

5. 发明授权

US06627618B2 Materials and methods for intracellular delivery of biologically active molecules 失效
标题翻译：用于细胞内递送生物活性分子的材料和方法
公开(公告)号：US06627618B2
公开(公告)日：2003-09-30
申请号：US09755681
申请日：2001-01-05
申请人： Volkmar Weissig , Jeffrey Allen Hughes , Jürgen Lasch , Thomas Cardon Rowe
发明人： Volkmar Weissig , Jeffrey Allen Hughes , Jürgen Lasch , Thomas Cardon Rowe
IPC分类号： A61K4800
CPC分类号： C12N15/88 , C12N15/87 , Y10S977/905 , Y10S977/907 , Y10S977/914 , Y10S977/916 , Y10S977/92
摘要： The subject invention finds utility in the area of gene therapy of diseases. More specifically, the invention concerns the making of a novel non-viral vector which can bind to desired DNA to form a combination useful to transfect diseased mitochondria of human or animal cells. The non-viral vector comprises a dequalinium salt subjected to standard liposome production procedures to obtain the vector named DQAsomes.
摘要翻译：本发明可用于疾病的基因治疗领域。更具体地说，本发明涉及可以结合所需DNA以形成可用于转染人或动物细胞的病态线粒体的组合的新型非病毒载体的制备。非病毒载体包含脱金属盐进行标准脂质体生产程序以获得称为DQAsomes的载体。

6. 发明授权

US06171863B2 Materials and methods for intracellular delivery of biologically active molecules 失效
标题翻译：用于细胞内递送生物活性分子的材料和方法
公开(公告)号：US06171863B2
公开(公告)日：2001-01-09
申请号：US09148953
申请日：1998-09-08
申请人： Volkmar Weissig , Jeffrey Allen Hughes , J{umlaut over (u)}rgen Lasch , Thomas Cardon Rowe
发明人： Volkmar Weissig , Jeffrey Allen Hughes , J{umlaut over (u)}rgen Lasch , Thomas Cardon Rowe
IPC分类号： C12N1588
CPC分类号： C12N15/88 , C12N15/87 , Y10S977/905 , Y10S977/907 , Y10S977/914 , Y10S977/916 , Y10S977/92
摘要： The subject invention finds utility in the area of gene therapy of diseases. More specifically, the invention concerns the making of a novel non-viral vector which can bind to desired DNA to form a combination useful to transfect diseased mitochondria of human or animal cells. The non-viral vector comprises a dequalinium salt subjected to standard liposome production procedures to obtain the vector names DQAsomes.
摘要翻译：本发明可用于疾病的基因治疗领域。更具体地说，本发明涉及可以结合所需DNA以形成可用于转染人或动物细胞的病态线粒体的组合的新型非病毒载体的制备。非病毒载体包含脱金属盐进行标准脂质体生产程序以获得载体名称DQAsomes。

7. 发明授权

US6090619A Materials and methods for intracellular delivery of biologically active molecules 失效
公开(公告)号：US6090619A
公开(公告)日：2000-07-18
申请号：US929175
申请日：1997-09-08
申请人： Volkmar Weissig , Jeffrey Allen Hughes , Jurgen Lasch , Thomas Cardon Rowe
发明人： Volkmar Weissig , Jeffrey Allen Hughes , Jurgen Lasch , Thomas Cardon Rowe
IPC分类号： C12N15/09 , C12N15/87 , C12N15/88 , C12N15/00 , A61K48/00
CPC分类号： C12N15/88 , C12N15/87 , Y10S977/905 , Y10S977/907 , Y10S977/914 , Y10S977/916 , Y10S977/92
摘要： The subject invention finds utility in the area of gene therapy of diseases. More specifically, the invention concerns the making of a novel non-viral vector which can bind to desired DNA to form a combination useful to transfect diseased mitochondria of human or animal cells. The non-viral vector comprises a dequalinium salt subjected to standard liposome production procedures to obtain the vector named DQAsomes.

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