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    METHODS FOR TREATING PARKINSON'S DISEASE
    53.
    发明申请
    METHODS FOR TREATING PARKINSON'S DISEASE 审中-公开

    公开(公告)号：WO2021119615A1

    公开(公告)日：2021-06-17

    申请号：PCT/US2020/064928

    申请日：2020-12-14

    Applicant: MEIRAGTX UK II LIMITED

    Inventor： FORBES, Alexandria ,  DURING, Matthew

    IPC: A61K35/12 ,  A61K38/51 ,  A61K48/00 ,  A61P25/00 ,  A61P25/14 ,  A61P25/16

    Abstract: Disclosed are methods for treating neurological disorders such as Parkinson's disease (PD) using glutamic acid decarboxylase (GAD) and identifying PD patients that will be most receptive to the method of treating PD. In one aspect, the disclosure provides a method of treating PD in a subject in need thereof, the method comprising: (a) identifying a subject having less than about 10 hours, and preferably less than about 8 hours, of on-time per day; and (b) administering to the subject a composition comprising a therapeutically effective amount of one or more vectors to the subthalamic nucleus of the patient, wherein each vector comprises a nucleic acid sequence encoding glutamic acid decarboxylase (GAD) and wherein the subject's on-time is increased.

    REGULATION OF GENE EXPRESSION THROUGH APTAMER-MODULATED POLYADENYLATION
    57.
    发明申请
    REGULATION OF GENE EXPRESSION THROUGH APTAMER-MODULATED POLYADENYLATION 审中-公开
    APTAMER调节聚腺苷酸化对基因表达的调控

    公开(公告)号：WO2017136591A1

    公开(公告)日：2017-08-10

    申请号：PCT/US2017/016279

    申请日：2017-02-02

    Applicant: MEIRAGTX UK II LIMITED ,  MEIRAGTX, LLC

    Inventor： VOLLES, Michael, J. ,  DANOS, Olivier, F. ,  GUO, Xuecui

    IPC: A61K48/00 ,  C12N15/86 ,  C07H21/04

    CPC classification number: A61K48/00 ,  C12N15/115 ,  C12N15/63 ,  C12N15/85 ,  C12N2310/16 ,  C12N2830/42

    Abstract: The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of U1 small nuclear ribonucleoprotein (snRNP)-mediated suppression of polyadenylation and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide construct contains a U1 binding site in the context of a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs.

    Abstract translation: 本发明提供了用于通过基于适体的U1小核核糖核蛋白（snRNP）介导的多聚腺苷酸化抑制来调节基因表达的多核苷酸构建体，以及使用所述构建体调节基因表达以响应 存在或不存在结合适体的配体。 多核苷酸构建体在包含效应子区和适体的核糖开关的上下文中含有U1结合位点，使得当适体结合配体时，发生靶基因表达。

    MODIFIED PROTEIN ENCODING SEQUENCES HAVING INCREASED RARE HEXAMER CONTENT
    58.
    发明申请
    MODIFIED PROTEIN ENCODING SEQUENCES HAVING INCREASED RARE HEXAMER CONTENT 审中-公开
    修饰的蛋白质编码序列具有增加的稀有六聚体含量

    公开(公告)号：WO2017079750A1

    公开(公告)日：2017-05-11

    申请号：PCT/US2016/060840

    申请日：2016-11-07

    Applicant: THE RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK

    Inventor： FUTCHER, Bruce ,  GARDIN, Justin ,  SKIENA, Steve ,  YUROVSKY, Alisa ,  WIMMER, Eckard ,  MUELLER, Steffen

    IPC: A61K39/12 ,  C12N7/04 ,  A61P31/12

    CPC classification number: C12N7/00

    Abstract: This invention provides a modified protein encoding sequence containing nucleotide substitutions at multiple locations in the protein encoding sequence, wherein the substitutions introduce rare hexamers. These hexamers may be Frame Dependent, or depleted in only the reading frame, or Frame Independent, or depleted in all three frames. Modified protein encoding sequences of the present invention may include modified viruses useful for vaccines.

    Abstract translation: 本发明提供了在蛋白质编码序列中的多个位置含有核苷酸取代的修饰的蛋白质编码序列，其中所述置换引入稀有六聚体。 这些六聚体可以是依赖于框架的，或者仅在阅读框架中消耗，或者与框架无关，或者在所有三个框架中耗尽。 本发明的修饰蛋白质编码序列可以包括用于疫苗的修饰病毒。

    REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED MODULATION OF ALTERNATIVE SPLICING
    59.
    发明申请
    REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED MODULATION OF ALTERNATIVE SPLICING 审中-公开
    通过APTAMER媒介调节替代性分离的基因表达调控

    公开(公告)号：WO2016126747A1

    公开(公告)日：2016-08-11

    申请号：PCT/US2016/016234

    申请日：2016-02-02

    Applicant: MEIRAGTX UK LIMITED

    Inventor： BOYNE, Alex, R. ,  DANOS, F., Olivier ,  VOLLES, J., Michael ,  GUO, Xuecui

    IPC: C12N15/11 ,  C12N15/63 ,  C12N15/85

    CPC classification number: C12N15/86 ,  A61K9/0048 ,  A61K38/44 ,  A61K48/0033 ,  A61K48/0066 ,  A61K48/0075 ,  C12N9/003 ,  C12N15/115 ,  C12N15/63 ,  C12N2310/16 ,  C12N2310/3519 ,  C12N2320/32 ,  C12N2320/33 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2840/002 ,  C12N2840/44 ,  C12Y105/01003

    Abstract: The invention provides a platform and methods of using the platform for the regulation of the expression of a target gene using exposure to an aptamer ligand (for example, a small molecule). The platform features a polynucleotide gene regulation cassette that is placed in the target gene and includes a synthetic riboswitch positioned in the context of a 5' intron-alternative exon-3' intron. The riboswitch comprises an effector region and a sensor region (e.g., an aptamer that binds a small molecule ligand) such that the alternative exon is spliced into the target gene mRNA when the ligand is not present thereby preventing expression of the target gene. When the ligand is present, the alternative exon is not spliced into the target gene mRNA thereby providing expression of the target gene.

    Abstract translation: 本发明提供了一种使用该平台来调节靶基因表达的平台和方法，该靶基因暴露于适体配体（例如小分子）。 该平台具有放置在靶基因中的多核苷酸基因调控盒，并且包括位于5'内含子替代外显子-3'内含子的上下文中的合成核糖开关。 核糖开关包括效应区和传感器区（例如，结合小分子配体的适体），使得当不存在配体时，替代外显子被剪接到靶基因mRNA中，从而防止靶基因的表达。 当存在配体时，替代的外显子不被剪接到靶基因mRNA中，从而提供靶基因的表达。

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