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    Compositions and Methods for the Treatment of Parkinson Disease by the Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
    2.
    发明申请
    Compositions and Methods for the Treatment of Parkinson Disease by the Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types 审中-公开
    通过将寡核苷酸分子选择性递送至特定神经元类型来治疗帕金森病的组合物和方法

    公开(公告)号：US20160083726A1

    公开(公告)日：2016-03-24

    申请号：US14803811

    申请日：2015-07-20

    Applicant: nLife Therapeutics, S.L.

    Inventor： Andrés Pablo MONTEFELTRO ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Pérez ,  Miquel Vila Bover ,  Maria del Carmen Carmona Orozco

    IPC: C12N15/113

    CPC classification number: C12N15/113 ,  A61K31/7105 ,  A61K47/55 ,  C12N2310/14

    Abstract: The invention provides a conjugate comprising (i) a selectivity agent which binds specifically to one or more neurotransmitter transporters selected from the group consisting of a dopamine transporter (DAT), serotonin transporter (SERT) or a norepinephrine transporter (NET) and (ii) a nucleic acid capable of specifically binding to a target molecule which is expressed in the same cell as the neurotransmitter transporter wherein said target molecule is α-synuclein or the mRNA encoding α-synuclein. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interest and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.

    Abstract translation: 本发明提供了一种缀合物，其包含（i）特异性结合一种或多种选自多巴胺转运蛋白（DAT），血清素转运蛋白（SERT）或去甲肾上腺素转运蛋白（NET））的神经递质转运蛋白的选择剂，和（ii） 能够特异性结合到与神经递质转运体相同的细胞中表达的靶分子的核酸，其中所述靶分子是α-突触核蛋白或编码α-突触核蛋白的mRNA。 本发明的缀合物可用于将核酸递送至感兴趣的细胞，因此用于治疗需要下调由靶核酸编码的蛋白质以及用于递送 成像剂到细胞用于诊断目的。

    Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
    3.
    发明授权
    Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types 有权
    通过将寡核苷酸分子选择性递送到特定神经元类型来治疗帕金森病的组合物和方法

    公开(公告)号：US09084825B2

    公开(公告)日：2015-07-21

    申请号：US14065024

    申请日：2013-10-28

    Applicant: nLife Therapeutics, S.L.

    Inventor： Andrés Pablo Montefeltro ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Pérez ,  Miquel Vila Bover ,  Maria del Carmen Carmona Orozco

    IPC: A61K47/48 ,  A61K31/7105

    CPC classification number: C12N15/113 ,  A61K31/7105 ,  A61K47/55 ,  C12N2310/14

    Abstract: The invention provides a conjugate comprising (i) a selectivity agent which binds specifically to one or more neurotransmitter transporters selected from the group consisting of a dopamine transporter (DAT), serotonin transporter (SERT) or a norepinephrine transporter (NET) and (ii) a nucleic acid capable of specifically binding to a target molecule which is expressed in the same cell as the neurotransmitter transporter wherein said target molecule is α-synuclein or the mRNA encoding α-synuclein. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interest and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.

    Abstract translation: 本发明提供了一种缀合物，其包含（i）特异性结合选自多巴胺转运蛋白（DAT），血清素转运蛋白（SERT）或去甲肾上腺素转运蛋白（NET））的一种或多种神经递质转运蛋白的选择剂，和（ii） 能够特异性结合到与神经递质转运体相同的细胞中表达的靶分子的核酸，其中所述靶分子是α-突触核蛋白或编码α-突触核蛋白的mRNA。 本发明的缀合物可用于将核酸递送至感兴趣的细胞，因此用于治疗需要下调由靶核酸编码的蛋白质以及用于递送 成像剂到细胞用于诊断目的。

    Compositions and Methods for the Treatment of Parkinson Disease by the Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
    6.
    发明申请
    Compositions and Methods for the Treatment of Parkinson Disease by the Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types 有权
    通过将寡核苷酸分子选择性递送至特定神经元类型来治疗帕金森病的组合物和方法

    公开(公告)号：US20140120158A1

    公开(公告)日：2014-05-01

    申请号：US14065024

    申请日：2013-10-28

    Applicant: nLife Therapeutics, S.L.

    Inventor： Andrés Pablo MONTEFELTRO ,  Gabriel Alvarado Urbina ,  Analia Bortolozzi Biassoni ,  Francesc Artigas Pérez ,  Miquel Vila Bover

    IPC: A61K47/48

    CPC classification number: C12N15/113 ,  A61K31/7105 ,  A61K47/55 ,  C12N2310/14

    Abstract: The invention provides a conjugate comprising (i) a selectivity agent which binds specifically to one or more neurotransmitter transporters selected from the group consisting of a dopamine transporter (DAT), serotonin transporter (SERT) or a norepinephrine transporter (NET) and (ii) a nucleic acid capable of specifically binding to a target molecule which is expressed in the same cell as the neurotransmitter transporter wherein said target molecule is α-synuclein or the mRNA encoding α-synuclein. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interest and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.

    Abstract translation: 本发明提供了一种缀合物，其包含（i）特异性结合一种或多种选自多巴胺转运蛋白（DAT），血清素转运蛋白（SERT）或去甲肾上腺素转运蛋白（NET））的神经递质转运蛋白的选择剂，和（ii） 能够特异性结合到与神经递质转运体相同的细胞中表达的靶分子的核酸，其中所述靶分子是α-突触核蛋白或编码α-突触核蛋白的mRNA。 本发明的缀合物可用于将核酸递送至感兴趣的细胞，因此用于治疗需要下调由靶核酸编码的蛋白质以及用于递送 成像剂到细胞用于诊断目的。

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