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    • 1. 发明申请
    • NOVEL COMPOUNDS FOR MODULATING NEOVASCULARISATION AND METHODS OF TREATMENT USING THESE COMPOUNDS
    • 用于调节新化合物的新型化合物和使用这些化合物的治疗方法
    • WO2011056073A3
    • 2011-12-22
    • PCT/NL2010050736
    • 2010-11-04
    • UNIV ERASMUS MEDICAL CTDUCKERS HENRICUS JOHANNES
    • DUCKERS HENRICUS JOHANNES
    • A61K38/16
    • A61K38/1709A61K38/177A61K48/00C12N15/113C12N2310/11C12N2310/14
    • The invention relates to a method for modulating neovascularisation of a tissue in a subject in need thereof, said method comprising administering to said subject a therapeutically effective amount of a compound or a combination of compounds selected from an isolated nucleic acid molecule comprising a gene selected from the group consisting of RIKEN c DNA 9430020K01, Agtrl1, Apelin, Stabilin 1, Stabilin 2, TNFaip8l1, TNFaip8 and FGD5, and homologues thereof; a gene product encoded by said genes, or encoded by homologues of these genes, and functional fragments thereof; an antibody or derivative thereof directed against a gene product of said genes, or encoded by homologues of these genes, and functional fragments thereof, said derivative preferably being selected from the group consisting of scFv fragments, Fab fragments, chimeric antibodies, bifunctional antibodies, intrabodies, and other antibody-derived molecules; an antisense molecule, in particular an antisense RNA or antisense oligodeoxynucleotide, an RNAi molecule (siRNA or mi RNA) or a ribozyme capable of binding under stringent hybridization conditions to a gene or an m RNA gene product of said genes and homologues thereof; a small molecule interfering with the biological activity of a gene product of said genes and homologues thereof, and a (glycol)protein, a hormone and other biologically active compounds capable of interacting with said genes and homologues thereof or with a gene product thereof.
    • 本发明涉及一种用于调节有需要的受试者的组织新血管形成的方法,所述方法包括向所述受试者施用治疗有效量的化合物或化合物组合,所述化合物或组合选自包含选自以下的基因的分离的核酸分子: 由RIKEN c DNA 9430020K01,Agtrl1,Apelin,Stabilin 1,Stabilin 2,TNFaip8I1,TNFaip8和FGD5组成的组及其同源物; 由所述基因编码或由这些基因的同源物编码的基因产物及其功能片段; 针对所述基因的基因产物或由这些基因的同系物编码的抗体或其衍生物及其功能片段,所述衍生物优选选自scFv片段,Fab片段,嵌合抗体,双功能抗体,胞内抗体 ,和其他抗体衍生的分子; 反义分子,特别是反义RNA或反义寡脱氧核苷酸,RNAi分子(siRNA或miRNA)或能够在严格杂交条件下与所述基因及其同源物的基因或m RNA基因产物结合的核酶; 干扰所述基因及其同系物的基因产物的生物学活性的小分子,以及能够与所述基因及其同系物或其基因产物相互作用的(二醇)蛋白质,激素和其他生物活性化合物。
    • 4. 发明申请
    • IMPROVED METHODS AND MEANS FOR LENTIVIRAL GENE DELIVERY
    • 改进的方法和手段用于LENIVEIRAL GENE DELIVERY
    • WO2008136670A3
    • 2009-02-19
    • PCT/NL2008050269
    • 2008-05-02
    • UNIV ERASMUS MEDICAL CTVAN TIL NICO PETERVERSTEGEN MONIQUE MARIA ANDREAWAGEMAKER GERARD
    • VAN TIL NICO PETERVERSTEGEN MONIQUE MARIA ANDREAWAGEMAKER GERARD
    • C12N15/09
    • C12N15/86C12N2740/16043
    • The invention relates to the field of gene therapy and more in specific to lentiviral gene delivery vehicles and methods for efficient transduction of lentiviral gene delivery vehicles into hematopoietic stem cells and their descendants. Preferably, the invention provides in one of its embodiments a method of gene transfer into e.g. pluripotent hematopoietic stem cells and their descendants, enabling successful transduction of cells, including transplantable cell populations comprising hematopoietic stem cells that give rise to progeny expressing the transduced gene(s). The invention further comprises a method for treating a variety of hereditary and acquired human disease by transfer of therapeutically active genes into hematopoietic stem cells. As a non-limiting example, the invention shows that symptoms associated with Pompe disease are (completely) reduced and/or alleviated by treatment of a subject suffering from Pompe disease withJiematopoietic stem cell transduced with an alpha- glucosidase comprising lentiviral vector.
    • 本发明涉及基因治疗领域,更具体涉及慢病毒基因递送载体以及用于将慢病毒基因递送载体有效转导至造血干细胞及其后代的方法。 优选地,本发明在其一个实施方案中提供了将基因转移到例如 多能造血干细胞及其后代,能够成功地转导细胞,包括可引起表达转导基因的后代的造血干细胞的可移植细胞群。 本发明还包括通过将治疗活性基因转移至造血干细胞来治疗各种遗传和获得性人类疾病的方法。 作为非限制性实例,本发明表明,通过用包含慢病毒载体的α-葡糖苷酶转导的造血干细胞治疗患有庞培病的受试者,(完全)减少和/或减轻与庞皮病相关的症状。