Retinol binding protein and transthyretin modulators for treating hypertosis, Alzheimer's disease or idiopathic intracranial hypertension
申请人:ReVision Therapeutics, Inc.
公开号:EP2289500A1
公开(公告)日:2011-03-02
Described herein are methods and compositions for treating certain retinol-related diseases and conditions by modulation of transthyretin (TTR) and retinol binding protein (RBP) availability in the subject. For example, the methods and compositions provide for therapeutic agents for the treatment and/or prevention of age-related macular degeneration and/or dystrophies, metabolic disorders, idiopathic intracranial hypertension, hyperostosis, and protein misfolding and aggregation diseases. The compositions disclosed may be used as single agent therapy or in combination with other agents or therapies. In addition, described herein are methods and assays for selecting appropriate agents that can modulate the TTR and RBP availability in a subject.
本文描述了通过调节受试者体内转甲状腺素(TTR)和视黄醇结合蛋白(RBP)的可用性来治疗某些视黄醇相关疾病和病症的方法和组合物。例如,这些方法和组合物提供了治疗和/或预防老年性黄斑变性和/或营养不良、代谢紊乱、特发性颅内高压、骨质疏松症以及蛋白质错误折叠和聚集疾病的治疗剂。所公开的组合物可用作单剂疗法,也可与其他制剂或疗法联合使用。此外,本文还描述了用于选择可调节受试者体内 TTR 和 RBP 可用性的适当制剂的方法和检测方法。