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    MITOCHONDRIAL TARGETING AND THERAPEUTIC USE THEREOF
    3.
    发明申请
    MITOCHONDRIAL TARGETING AND THERAPEUTIC USE THEREOF 有权
    麻醉指导和治疗方法

    公开(公告)号：US20160237129A1

    公开(公告)日：2016-08-18

    申请号：US14993500

    申请日：2016-01-12

    申请人： Shire Human Genetic Therapies, Inc.

    发明人： Dennis Keefe ,  Michael Concino ,  Michael Heartlein ,  Serene Josiah ,  Bettina Strack-Logue

    IPC分类号： C07K14/47

    CPC分类号： C07K14/47 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K38/00 ,  A61K38/1709 ,  A61K47/645 ,  A61K49/0008 ,  C07K7/08 ,  C07K2319/07 ,  C07K2319/10

    摘要： The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.

    摘要翻译： 本发明尤其提供了用于治疗弗里德里希共济失调的组合物和方法，其基于可以替代天然FXN蛋白活性或拯救一种或多种与frataxin缺陷相关的表型或症状的线粒体的治疗性部分的有效靶向。 在一些实施方案中，本发明提供靶向治疗剂，其包含治疗部分，其为具有N末端和C末端的多肽，与N末端的治疗部分相关的线粒体靶向序列，以及线粒体膜 与C-末端的治疗部分相关的穿透肽，其中治疗部分在细胞进入时靶向线粒体。

    Antisense oligonucleotide modulators of serotonin receptor 2C and uses thereof
    5.
    发明授权
    Antisense oligonucleotide modulators of serotonin receptor 2C and uses thereof 有权
    5-羟色胺受体2C的反义寡核苷酸调节剂及其用途

    公开(公告)号：US09567585B2

    公开(公告)日：2017-02-14

    申请号：US14357388

    申请日：2012-11-09

    申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

    发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

    IPC分类号： C12N15/11 ,  C12N15/113

    CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/33

    摘要： The present invention provides, among other things, oligonucleotide modulators of human 5′-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.

    摘要翻译： 本发明尤其提供了人5-HT 2C受体（HTR2C）的寡核苷酸调节剂以及用于治疗基于这些调节剂的HTR2C相关疾病，病症或病症的改进的方法和组合物。 特别地，根据本发明的寡核苷酸调节剂靶向人HTR2C前mRNA的外显子V /内含子V结中的特异性区域并驱动HTR2C Vb剪接同种型的表达，导致未编辑的强HTR2C受体和增强的5-羟色胺的产生增加 受体活性。

    ANTI-FLT-1 ANTIBODIES IN TREATING DUCHENNE MUSCULAR DYSTROPHY
    6.
    发明申请
    ANTI-FLT-1 ANTIBODIES IN TREATING DUCHENNE MUSCULAR DYSTROPHY 有权
    抗-FTT-1抗体治疗杜氏肌肉晶状体

    公开(公告)号：US20150361174A1

    公开(公告)日：2015-12-17

    申请号：US14763881

    申请日：2014-01-28

    申请人： SHIRE HUMAN GENETIC THERAPIES, INC.

    发明人： Serene Josiah ,  Thomas M. Luby ,  Atsushi Asakura ,  Dennis Keefe ,  Lawrence Charnas

    IPC分类号： C07K16/28

    CPC分类号： C07K16/2863 ,  A61K2039/505 ,  C07K16/2866 ,  C07K2317/33 ,  C07K2317/54 ,  C07K2317/55 ,  C07K2317/76

    摘要： The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

    摘要翻译： 本发明尤其提供了用于治疗肌营养不良的方法和组合物，特别是杜烯肌营养不良症（DMD）。 在一些实施方案中，根据本发明的方法包括对患有或易感DMD的个体施用有效量的抗Flt-1抗体或其抗原结合片段，使得至少一种症状或特征 的DMD强度，严重程度或频率降低，或延迟发作。

    ANTISENSE OLIGONUCLEOTIDE MODULATORS OF SEROTONIN RECEPTOR 2C AND USES THEREOF
    10.
    发明申请
    ANTISENSE OLIGONUCLEOTIDE MODULATORS OF SEROTONIN RECEPTOR 2C AND USES THEREOF 有权
    血清素受体2C的抗体寡核苷酸调节因子及其用途

    公开(公告)号：US20140275222A1

    公开(公告)日：2014-09-18

    申请号：US14357388

    申请日：2012-11-09

    申请人： Shire Human Genetic Therapies, Inc. ,  University of Kentucky

    发明人： Stefan Stamm ,  Manli Shen ,  Serene Josiah

    IPC分类号： C12N15/113

    CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/33

    摘要： The present invention provides, among other things, oligonucleotide modulators of human 5′-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.

    摘要翻译： 本发明尤其提供了人5-HT 2C受体（HTR2C）的寡核苷酸调节剂以及用于治疗基于这些调节剂的HTR2C相关疾病，病症或病症的改进的方法和组合物。 特别地，根据本发明的寡核苷酸调节剂靶向人HTR2C前mRNA的外显子V /内含子V结中的特异性区域并驱动HTR2C Vb剪接同种型的表达，导致未编辑的强HTR2C受体和增强的5-羟色胺的产生增加 受体活性。