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    • 5. 发明申请
    • METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND TREATMENT OF PROLIFERATIVE DISORDERS
    • 用于诊断和治疗增生性疾病的方法和组合物
    • WO2009149166A2
    • 2009-12-10
    • PCT/US2009046103
    • 2009-06-03
    • CHILDRENS HOSP MEDICAL CENTERRATNER NANCYMILLER SHYRA J
    • RATNER NANCYMILLER SHYRA J
    • C12Q1/68
    • G01N33/57407C12Q1/6886C12Q2600/158G01N2500/04G01N2800/52
    • The present invention identifies expression markers associated with neurofibromatosis 1 (NF1) by examining gene expression in tissue from lesions from neurofibromas, plexiform neurofibromas and malignant peripheral nerve sheath tumors (MPNST). The present invention also provides for diagnostic markers as well as markers that can be used to monitor disease states, disease progression, drug toxicity, drug efficacy and drug metabolism. The present invention relates to novel, rapid, reliable and effective assays for screening and identifying pharmaceutically effective compounds that act as a therapeutic agent for the treatment of a proliferative disorder. In another aspect, the present invention provides a method of treating a patient with neurofibromatosis and/or MPNST, comprising administering to the patient a pharmaceutical composition, wherein the composition alters the expression of at least one gene related to the NF1-Ras pathway.
    • 本发明通过检查来自神经纤维瘤,丛状神经纤维瘤和恶性周围神经鞘瘤(MPNST)病变的组织中的基因表达来鉴定与神经纤维瘤病1(NF1)相关的表达标志物。 本发明还提供诊断标志物以及可用于监测疾病状态,疾病进展,药物毒性,药物功效和药物代谢的标志物。 本发明涉及用于筛选和鉴定用作治疗增殖性疾病的治疗剂的药学有效化合物的新型,快速,可靠和有效的测定。 在另一方面,本发明提供了治疗患有神经纤维瘤病和/或MPNST的患者的方法,包括向患者施用药物组合物,其中所述组合物改变与NF1-Ras途径相关的至少一种基因的表达。