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    BIGLYCAN AND RELATED THERAPEUTICS AND METHODS OF USE
    1.
    发明申请
    BIGLYCAN AND RELATED THERAPEUTICS AND METHODS OF USE 审中-公开
    BIGLYCAN及相关的治疗方法和使用方法

    公开(公告)号：WO0136475A9

    公开(公告)日：2002-11-21

    申请号：PCT/US0031661

    申请日：2000-11-17

    Applicant: UNIV BROWN RES FOUND

    Inventor： FALLON JUSTIN ,  MCKECHNIE BETH ,  RAFII MICHAEL ,  CREELY HILLIARY ,  BOWE MARK ,  FERRI RAYMOND

    IPC: A61K38/17 ,  G01N33/68 ,  C07K14/47 ,  C07K14/46 ,  C12P21/02 ,  G01N33/50

    CPC classification number: A61K38/1709 ,  G01N33/6887 ,  G01N2400/12 ,  G01N2800/2878 ,  G01N2800/2885

    Abstract: The invention provides compositions and methods for treating, preventing, and diagnosing diseases or conditions associated with an abnormal level or activity of biglycan; disorders associated with an unstable cytoplasmic membrane, due, e.g., to an unstable dystrophin associated protein complex (DAPC); disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation. Example of diseases include muscular dystrophies, such as Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, neuromuscular disorders and neurological disorders.

    Abstract translation: 本发明提供了用于治疗，预防和诊断与双糖链蛋白聚糖异常水平或活性相关的疾病或病症的组合物和方法; 由于例如不稳定的肌营养不良蛋白复合体（DAPC）而与不稳定的细胞质膜相关的病症; 与异常突触或神经肌肉接头有关的疾病，包括由异常MuSK激活或乙酰胆碱受体（AChR）聚集引起的疾病。 疾病的实例包括肌营养不良，例如杜兴氏肌营养不良，贝克氏肌营养不良，神经肌肉疾病和神经障碍。

    METHOD FOR DIAGNOSING AND TREATING MUSCULAR DYSTROPHY
    5.
    发明申请
    METHOD FOR DIAGNOSING AND TREATING MUSCULAR DYSTROPHY 审中-公开
    用于诊断和治疗肌肉晶状体的方法

    公开(公告)号：WO2010125358A1

    公开(公告)日：2010-11-04

    申请号：PCT/GB2010/000874

    申请日：2010-04-30

    Applicant: THE UNIVERSITY OF BIRMINGHAM ,  SMITH, Janet ,  MERRICK, Deborah

    Inventor： SMITH, Janet ,  MERRICK, Deborah

    IPC: G01N33/68 ,  A61K48/00

    CPC classification number: G01N33/6893 ,  A61K38/177 ,  G01N2800/2878 ,  G01N2800/2885

    Abstract: The invention describes a method for identifying an individual exhibiting symptoms of, or having a propensity to develop muscular dystrophy comprising determining the level of expression in a tissue sample from the individual of one or more proteins selected from pax-7, caveolin-3 and/or fast-myosin. The use of caveolin-3 to treat muscular dystrophy and compositions containing the compound are also claimed.

    Abstract translation: 本发明描述了一种用于鉴定展现肌肉营养不良症状或具有发展肌营养不良倾向的个体的方法，包括确定来自个体的组织样品中的一种或多种蛋白质的表达水平，所述蛋白质选自pax-7，caveolin-3和/ 或快肌球蛋白。 还要求使用窖蛋白-3来治疗肌营养不良症和含有该化合物的组合物。

    METHODS FOR IDENTIFYING COMPOUNDS FOR REGULATING MUSCLE MASS OR FUNCTION USING DOPAMINE RECEPTORS
    6.
    发明申请
    METHODS FOR IDENTIFYING COMPOUNDS FOR REGULATING MUSCLE MASS OR FUNCTION USING DOPAMINE RECEPTORS 审中-公开
    用于鉴定使用多巴胺受体调节肌肉质量或功能的化合物的方法

    公开(公告)号：WO2005017484A3

    公开(公告)日：2007-05-31

    申请号：PCT/US0322588

    申请日：2003-07-18

    Applicant: PROCTER & GAMBLE

    Inventor： ISFORT ROBERT JOSEPH ,  SHELDON RUSSELL JAMES

    IPC: G01N33/53 ,  A61K31/55 ,  A61K49/00 ,  C07K16/28 ,  G01N33/566 ,  G01N33/567 ,  G01N33/68 ,  G01N33/94

    CPC classification number: C07K16/28 ,  A61K31/55 ,  C07K2317/21 ,  G01N33/6887 ,  G01N33/9413 ,  G01N2500/00 ,  G01N2800/2878 ,  G01N2800/2885

    Abstract: Screening methods for identifying compounds that bind to or activate (D 1 or D 5 dopamine receptors individually or in combination) or regulate or potentially regulate skeletal muscle mass or function in vivo. Also disclosed are screening methods for identifying compounds that prolong or augment the activation of D 1 or D 5 dopamine receptors or of D 1 or D 5 dopamine receptor signal transduction pathways and increase D 1 or D 5 dopamine receptor expression. Pharmaceutical compositions comprising D 1 or D 5 dopamine receptor agonists, antibodies to D 1 or D 5 dopamine receptors and methods for increasing skeletal muscle mass or function or for the treatment of skeletal muscle atrophy using D 1 or D 5 dopamine receptors as the target for intervention and methods for treatment of muscular dystrophies are described.

    Abstract translation: 用于鉴定结合或活化（D 1 N或D 5多巴胺受体单独或组合）的化合物的筛选方法或调节或潜在地调节骨骼肌质量或体内功能。 还公开了用于鉴定延长或增加D 1 N或D 5 N多巴胺受体或D 1 N或D 3多巴胺受体的活化的化合物的筛选方法， 多巴胺受体信号转导途径，并增加D 1 N或D 5多巴胺受体表达。 包含D 1或D 5 N多巴胺受体激动剂的药物组合物，D 1或D 5多巴胺受体的抗体和 用于增加骨骼肌质量或功能的方法或使用D 1或D 5多巴胺受体作为干预目标和用于治疗肌营养不良症的方法来治疗骨骼肌萎缩的方法 被描述。

    PROBES FOR AND METHODS OF DIAGNOSIS FOR MUSCULAR DYSTROPHY
    7.
    发明申请
    PROBES FOR AND METHODS OF DIAGNOSIS FOR MUSCULAR DYSTROPHY 审中-公开
    探针和方法的诊断为肌肉样本

    公开(公告)号：WO1989006286A2

    公开(公告)日：1989-07-13

    申请号：PCT/US1988004504

    申请日：1988-12-16

    Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION

    Inventor： THE CHILDREN'S MEDICAL CENTER CORPORATION ,  KUNKEL, Louis, M. ,  MONACO, Anthony ,  HOFFMAN, Eric, P. ,  KOENIG, Michel

    IPC: C12Q01/68

    CPC classification number: G01N33/6896 ,  C12Q1/6883 ,  C12Q2600/156 ,  G01N33/6887 ,  G01N2800/2878 ,  G01N2800/2885

    Abstract: The invention relates to a muscular dystrophy (MD) probe comprising a substantially purified single-stranded nucleic acid sequence capable of hybridizing to a region of DNA on a human X chromosome between the deletion break point at Xp21.3 and the translocation break point at X;11. The invention also relates to a 14kb cDNA corresponding to the complete MD gene and probes produced therefrom useful in genetic methods of diagnosis of MD. Furthermore, the invention relates to the polypeptide, dystrophin, which corresponds to the MD gene product, and antibodies thereto that are useful in a variety of methods for immunodiagnosis of MD.

    Abstract translation: 本发明涉及一种肌营养不良症（MD）探针，其包含基本上纯化的单链核酸序列，其能够与人X染色体上的DNA区域在Xp21.3的缺失断点和X位移断裂点之间杂交 ; 11。 本发明还涉及对应于完整MD基因的14kb cDNA和由其产生的探针，其可用于诊断MD的遗传方法。 此外，本发明涉及对应于MD基因产物的多肽，肌营养不良蛋白及其抗体，其可用于多种免疫诊断MD的方法。

    DIAGNOSIS OF AUTOSOMAL MUSCULAR DYSTROPHY
    9.
    发明申请
    DIAGNOSIS OF AUTOSOMAL MUSCULAR DYSTROPHY 审中-公开
    诊断自动肌肉晶状体

    公开(公告)号：WO1994007143A1

    公开(公告)日：1994-03-31

    申请号：PCT/US1993008585

    申请日：1993-09-13

    Applicant: UNIVERSITY OF IOWA RESEARCH FOUNDATION

    Inventor： UNIVERSITY OF IOWA RESEARCH FOUNDATION ,  CAMPBELL, Kevin, P. ,  MATSUMURA, Kiichiro

    IPC: G01N33/68

    CPC classification number: C07K14/4708 ,  A61K38/00 ,  A61K39/00 ,  C07K14/4707 ,  C07K14/705 ,  C07K16/18 ,  C07K16/28 ,  C07K2317/34 ,  G01N33/6887 ,  G01N33/6896 ,  G01N2800/2878 ,  G01N2800/2885 ,  Y10S435/96 ,  Y10S436/811

    Abstract: Disclosed are methods for the diagnosis of autosomal muscular dystrophy through the analysis of muscle tissue using antibodies reactive with components of the dystrophin-glycoprotein complex. An experimental muscle tissue sample, treated if necessary to render components of the dystrophin-glycoprotein complex available for antibody binding, is contacted with an antibody which binds to a dystrophin-associated protein. The extent of antibody binding is determined, and compared to the extent of antibody binding to normal control tissue. A substantial reduction in the extent of binding to experimental tissue, as compared with normal control tissue, being diagnostic of autosomal muscular dystrophy. Among the autosomal muscular dystrophies which are detectable by the methods described herein are Fukuyama muscular dystrophy and severe childhood autosomal recessive muscular dystrophy.

    Abstract translation: 公开了通过使用与肌营养不良蛋白 - 糖蛋白复合物的组分反应的抗体来分析肌肉组织来诊断常染色体肌营养不良的方法。 如果需要处理以使得可用于抗体结合的抗肌萎酸蛋白 - 糖蛋白复合物的组分被治疗的实验性肌肉组织样品与结合于肌营养不良蛋白相关蛋白的抗体接触。 测定抗体结合的程度，并与抗体与正常对照组织结合的程度进行比较。 与正常对照组织相比，结合实验组织的程度显着降低，是常染色体肌营养不良症的诊断。 通过本文所述的方法可检测的常染色体肌营养不良症是福山肌营养不良症和严重的儿童常染色体隐性遗传性肌营养不良症。

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