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    • 5. 发明申请
    • Mitochondriotropic Phospholipid Vesicles
    • 线粒体磷脂囊泡
    • US20080095834A1
    • 2008-04-24
    • US11885419
    • 2006-03-02
    • Volkmar WeissigSarathi BoddapatiRobert HansonVladimir Torchilin
    • Volkmar WeissigSarathi BoddapatiRobert HansonVladimir Torchilin
    • A61K9/127A61K31/70A61P43/00A61K38/00
    • A61K9/1272
    • 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.
    • 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。
    • 6. 发明授权
    • Composition for delivery of a mitochondrial genome to a cell
    • 用于将线粒体基因组递送至细胞的组合物
    • US07279326B2
    • 2007-10-09
    • US10485407
    • 2002-07-31
    • Volkmar WeissigVladimir Torchilin
    • Volkmar WeissigVladimir Torchilin
    • C12N15/00
    • A61K48/00C12N15/87
    • 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.
    • 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。