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    • 45. 发明申请
    • NOVEL TARGET GENES FOR DISEASES OF THE HEART
    • 心脏疾病的新目标基因
    • WO0192567A3
    • 2003-02-13
    • PCT/EP0106165
    • 2001-05-30
    • MEDIGENE AGBUNK DANIELAREUNER BIRGITBECK JOACHIMHENKEL THOMAS
    • BUNK DANIELAREUNER BIRGITBECK JOACHIMHENKEL THOMAS
    • A01K67/027A61K38/00A61K45/00A61K48/00A61P9/00A61P9/02A61P9/04A61P9/10A61P9/12C07K14/47C12N15/09C12N15/85C12Q1/50C12Q1/68G01N33/15G01N33/50G01N33/53G01N33/566A61K49/00C07K16/18
    • C12N15/8509A01K2217/05A01K2217/075A01K2267/03A01K2267/0375C07K14/47C12Q1/6883C12Q2600/136C12Q2600/158
    • The present invention relates to a variety of genes abnormally expressed in heart tissue as well as to fragments of such genes. Assessment of the expression level of these genes may be used for testing the predisposition of mammals and preferably humans for a heart disease or for an acute state of such a disease. Preferred diseases in accordance with the invention are congestive heart failure, dilative cardiomyopathy, hypertrophic cardiomyopathy and ischemic cardiomyopathy. The present invention further relates to methods of identifying compounds capable of normalizing the expression level of the aforementioned genes and of further genes affected by the abnormal expression. The identified compounds may be used for formulating compositions, preferably pharmaceutical compositions for preventing or treating diseases. They may also be used as lead compounds for the development of medicaments having an improved efficiency, a longer half-life, a decreased toxicity etc. and to be employed in the treatment of heart diseases. Included in the invention are also somatic gene therapy methods comprising the introduction of at least one functional copy of any of the above-mentioned genes into a suitable cell. Finally, the invention relates to non-human transgenic animals comprising at least one of the aforementioned genes in their germ line. The transgenic animals of the invention may be used for the development of medicaments for the treatment of heart diseases.
    • 本发明涉及在心脏组织中异常表达的各种基因以及这些基因的片段。 这些基因的表达水平的评估可以用于测试哺乳动物和优选人心脏病或这种疾病的急性状态的倾向。 根据本发明的优选疾病是充血性心力衰竭,扩张性心肌病,肥厚性心肌病和缺血性心肌病。 本发明还涉及鉴定能够使上述基因的表达水平正常化的化合物和受异常表达影响的其它基因的方法。 鉴定的化合物可用于配制组合物,优选用于预防或治疗疾病的药物组合物。 它们也可以用作开发具有改善的效率,更长的半衰期,降低的毒性等等并且用于治疗心脏病的药物的铅化合物。 本发明还包括体细胞基因治疗方法,其包括将任何上述基因的至少一个功能拷贝引入合适的细胞中。 最后,本发明涉及在其种系中包含至少一个上述基因的非人转基因动物。 本发明的转基因动物可用于开发用于治疗心脏病的药物。