公开(公告)号：US20050112121A1

公开(公告)日：2005-05-26

申请号：US10781060

申请日：2004-02-17

申请人： Spyridon Artavanis-Tsakonas ,  Richard Fehon ,  Panayiotis Zagouras ,  Christine Blaumueller

发明人： Spyridon Artavanis-Tsakonas ,  Richard Fehon ,  Panayiotis Zagouras ,  Christine Blaumueller

IPC分类号： A01N37/18 ,  A61K38/00 ,  A61K38/17 ,  A61K39/395 ,  C07K14/435 ,  C07K14/705 ,  C12N15/62 ,  G01N33/574

CPC分类号： C07K14/43581 ,  C07K14/705 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/20 ,  C07K2319/23 ,  C07K2319/70 ,  C07K2319/95 ,  C12N15/62

摘要： The present invention relates to therapeutic and diagnostic methods and compositions based on Notch proteins and nucleic acids. The invention provides for treatment of disorders of cell fate or differentiation by administration of a therapeutic compound of the invention. Such therapeutic compounds (termed herein “Therapeutics”) include Notch proteins and analogs and derivatives (including fragments) thereof, antibodies thereto, nucleic acids encoding the Notch proteins, analogs, or derivatives, Notch antisense nucleic acids, as well as toporythmic proteins and derivatives which bind to or otherwise interact with Notch proteins and their encoding nucleic acids and antibodies. In a preferred embodiment, a Therapeutic of the invention is administered to treat a cancerous condition, or to prevent progression from a pre-neoplastic or non-malignant state into a neoplastic or a malignant state. In other embodiments, a Therapeutic is administered to treat a nervous system disorder or to promote tissue regeneration and repair. In one embodiment, Therapeutics which antagonize, or inhibit, Notch function (hereinafter “Antagonist Therapeutics”) are administered for therapeutic effect. In another embodiment, Therapeutics which promote Notch function (hereinafter “Agonist Therapeutics”) are administered for therapeutic effect. Diagnostic methods and methods of inhibiting Notch expression are also provided.

摘要翻译： 本发明涉及基于Notch蛋白和核酸的治疗和诊断方法和组合物。 本发明提供了通过施用本发明的治疗化合物治疗细胞命运或分化的病症。 此类治疗化合物（本文称为“治疗学”）包括Notch蛋白及其类似物和衍生物（包括其片段），其抗体，编码Notch蛋白，类似物或衍生物的核酸，Notch反义核酸以及拓扑蛋白和衍生物 其与Notch蛋白质及其编码核酸和抗体结合或以其它方式相互作用。 在优选的实施方案中，施用本发明的治疗剂以治疗癌症状况，或预防从肿瘤前或非恶性状态进展为肿瘤或恶性状态。 在其它实施方案中，施用治疗剂以治疗神经系统疾病或促进组织再生和修复。 在一个实施方案中，施用拮抗或抑制Notch功能的治疗剂（以下称为“拮抗剂治疗剂”）用于治疗效果。 在另一个实施方案中，施用促进Notch功能的治疗剂（以下称为“激动剂治疗剂”）用于治疗效果。 还提供了抑制Notch表达的诊断方法和方法。