CELL PENETRATING TRANSPOSASE
    2.
    发明申请

    公开(公告)号:WO2020169673A1

    公开(公告)日:2020-08-27

    申请号:PCT/EP2020/054371

    申请日:2020-02-19

    IPC分类号: C12N15/90

    摘要: The Sleeping Beauty (SB) transposon is an efficient non-viral tool for inserting transgenes into cells. Its broad utilization in gene therapy has been hampered by uncontrolled transposase gene activity and the inability to use transposase protein directly. The present invention concerns the finding that SB transposase spontaneously penetrates mammalian cells and can be delivered with transposon DNA to gene-modify various cell lines, embryonic, hematopoietic and induced pluripotent stem cells. The invention provides methods and compounds to apply the cell penetrating function of transposase in methods of genetically engineering cells as well as using the transposase as a shuttle for delivering cargo into a target cell or even into a target cell organelle. Genomic integration frequency can be titrated using the technology of the invention, which adds an additional layer of safety, opening opportunities for advanced applications in genetic engineering and gene therapy.

    PHARMACEUTICAL COMPOSITIONS FOR PREVENTION AND/OR TREATMENT OF INFECTIONS AND ANTIBACTERIAL-INDUCED DYSFUNCTIONS

    公开(公告)号:WO2019206781A1

    公开(公告)日:2019-10-31

    申请号:PCT/EP2019/060017

    申请日:2019-04-17

    摘要: The present invention relates to the field of therapeutics and, more in particular, to pharmaceutical compositions for the prevention and/or treatment of bacterial infections and antibacterial-induced dysfunctions. The compositions of the present invention demonstrate high species-specificity in inhibiting the growth of a small number of bacterial species, and most importantly are effective also against multi drug resistant (MDR) clinical isolate species. Interestingly, one of those combinations pairs a non- antibiotic drug, vanillin, with an antibiotic drug, spectinomycin, to demonstrate a surprisingly strong inhibitory effect on the growth of clinically relevant Gram-negative pathogenic and multi-drug resistant E. coli isolates. A second set of compounds combines the polymyxin colistin with loperamide, a rifamycin, or a macrolide. Importantly, this invention relates to combinations that enable narrow-spectrum antibacterial therapies, constituting a major effort of current and future drug development efforts in order to prevent major side effects of antibacterial strategies. This invention also relates to pharmaceutical combinations useful to prevent an adverse effect on the gut microbiome, induced by the use of antibacterial compounds.

    ARCHAEAL PYRROLYSYL TRNA SYNTHETASES FOR ORTHOGONAL USE
    5.
    发明申请
    ARCHAEAL PYRROLYSYL TRNA SYNTHETASES FOR ORTHOGONAL USE 审中-公开
    用于正交使用的原虫聚吡咯酰化合物

    公开(公告)号:WO2018069481A1

    公开(公告)日:2018-04-19

    申请号:PCT/EP2017/076140

    申请日:2017-10-13

    IPC分类号: C12P21/02 C12N15/11

    摘要: The invention relates to archaeal pyrrolysyl tRNA synthetases lacking a nuclear localization signal and/or comprising a nuclear export signal. The invention also relates to polynucleotides encoding said pyrrolysyl tRNA synthetases, eukaryotic cells comprising said polynucleotide and tRNA acylated by the pyrrolysyl tRNA synthetase or a polynucleotide encoding such tRNA, methods utilizing said cells for preparing polypeptides comprising unnatural amino acid residues, and kits useful in said methods.

    摘要翻译: 本发明涉及缺乏核定位信号和/或包含核输出信号的古细菌吡咯赖氨酰tRNA合成酶。 本发明还涉及编码所述吡咯赖氨酰tRNA合成酶的多核苷酸,包含所述多核苷酸和由吡咯赖氨酰tRNA合成酶酰化的tRNA或编码此类tRNA的多核苷酸的真核细胞,利用所述细胞制备包含非天然氨基酸残基的多肽的方法,以及可用于所述 方法

    VIRAL POLYPEPTIDE FRAGMENTS THAT BIND CELLULAR POL II C-TERMINAL DOMAIN (CTD) AND THEIR USES
    6.
    发明申请
    VIRAL POLYPEPTIDE FRAGMENTS THAT BIND CELLULAR POL II C-TERMINAL DOMAIN (CTD) AND THEIR USES 审中-公开
    结合细胞pol II C-端结构域(CTD)的病毒多肽片段及其用途

    公开(公告)号:WO2018007615A1

    公开(公告)日:2018-01-11

    申请号:PCT/EP2017/067144

    申请日:2017-07-07

    IPC分类号: C12N9/12 G06F19/16

    摘要: The present invention relates to in silico methods for identifying compounds which decrease or prevent the binding of the viral RNA-dependent RNA polymerase from the Orthomyxoviridae family or variant thereof, to its ligand, (preferably to cellular Pol II, more preferably to CTD), as well as methods of producing the identified compounds. The present invention also relates to a compounds identifiable and/or producible by said methods. The present invention also relates to antibodies directed against the binding site of the RNA-dependent RNA polymerase, to its ligand (in particular to cellular Pol II, in particular to CTD of Pol II) as well as nucleic acids encoding said antibodies and vectors comprising the nucleic acid. The present invention relates to a pharmaceutical composition producible according to said method, and/or comprising said compound, said antibody, said nucleic acid, or said vector. The present invention also relates to the use of said compound, said antibody, said nucleic acid, said vector or said pharmaceutical in treating, ameliorating, or preventing disease conditions caused by viral infections with viruses of the Orthomyxoviridae family.

    摘要翻译: 本发明涉及用于鉴定减少或防止病毒RNA依赖性RNA聚合酶从正粘病毒科或其变体与其变体结合的化合物的计算机方法, 配体(优选转化为细胞Pol II,更优选转化为CTD),以及生产所鉴定的化合物的方法。 本发明还涉及可通过所述方法鉴定和/或生产的化合物。 本发明还涉及针对所述RNA依赖性RNA聚合酶的结合位点的抗体,其配体(尤其是对细胞RNA聚合酶II,特别是RNA聚合酶II的CTD)以及核酸编码所述抗体和载体,其包括 核酸。 本发明涉及根据所述方法可产生的和/或包含所述化合物,所述抗体,所述核酸或所述载体的药物组合物。 本发明还涉及所述化合物,所述抗体,所述核酸,所述载体或所述药物在治疗,改善或预防由正粘病毒科病毒感染病毒引起的疾病中的用途。