COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALLY ACTIVE MOLECULES
申请人:Jadhav Vasant
公开号:US20100015218A1
公开(公告)日:2010-01-21
The present invention relates to novel compositions and methods for potentiating the activity of biologically active molecules in conjunction with one or more delivery vehicles and one or more carrier molecules. Specifically, the invention features the use of a carrier molecule in combination with a delivery vehicle and a biologically active molecule of interest to potentiate the activity of the biologically active molecule. The carrier molecule can be biologically inert, inactive, or attenuated; or can alternately be biologically active in the same or different manner than the biologically active molecule of interest. Specifically, the invention features novel particle forming delivery agents including cationic lipids, microparticles, and nanoparticles that are useful for delivering various biologically active molecules to cells in conjunction with a carrier molecule. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism that are delivered intracellularly in conjunction with a carrier molecule. In various embodiments, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, to relevant cells and/or tissues, such as in a subject or organism, in conjunction with one or more carrier molecules. Such novel cationic lipids, microparticles, nanoparticles and transfection agents that are used in conjunction with one or more carrier molecules are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism.
本发明涉及一种新型组合物和方法,用于增强生物活性分子的活性,结合一个或多个传递载体和一个或多个载体分子。具体而言,本发明涉及使用载体分子与传递载体和感兴趣的生物活性分子结合,以增强生物活性分子的活性。载体分子可以是生物惰性、不活跃或减弱的;或者可以是与生物活性分子以相同或不同的方式活跃的。具体而言,本发明涉及新型形成颗粒的传递剂,包括阳离子脂质、微粒和纳米粒子,它们对于传递各种生物活性分子到细胞中与载体分子结合是有用的。本发明还涉及组合物和使用方法,用于研究、诊断和治疗在主体或有机体中对基因表达和/或活性调节有反应的特征、疾病和情况,这些组合物和方法是与载体分子一起递送到细胞内的。在各种实施例中,本发明涉及新型阳离子脂质、微粒、纳米粒子和转染剂,它们有效地转染或递送生物活性分子,如抗体(例如单克隆、嵌合、人源化等)、胆固醇、激素、抗病毒药物、肽、蛋白质、化学治疗药物、小分子、维生素、辅因子、核苷、核苷酸、寡核苷酸、酶核酸、反义核酸、三链体寡核苷酸、2,5-A嵌合体、异构酶、适配体、诱饵和其类似物以及小核酸分子,如短干扰核酸(siNA)、短干扰RNA(siRNA)、双链RNA(dsRNA)、微RNA(miRNA)和短发夹RNA(shRNA)分子,递送到相关的细胞和/或组织中,例如在主体或有机体中,与一个或多个载体分子结合。这种与一个或多个载体分子一起使用的新型阳离子脂质、微粒、纳米粒子和转染剂是有用的,例如提供组合物以预防、抑制或治疗细胞、主体或有机体中的疾病、情况或特征。