methods for treating retinol-related diseases, macular degeneration or dystrophy, and type I or type II diabetes

摘要

METHODS FOR TREATING RETINOL-RELATED DISEASES, MACULAR DEGENERATION OR DISEASE, TYPE I OR TYPE II DIABETES Methods and compositions for treating certain retinol-related diseases and conditions by modulating the availability of transthyretin (TTR) and retinol binding protein are described herein. (RBP) in the individual. For example, the methods and compositions provide therapeutic agents for the treatment and / or prevention of macular degeneration and / or age-related dystrophies, metabolic disorders, idiopathic intracranial hypertension, hyperostosis, and malfolding and protein aggregation diseases. The described compositions may be used as single agent therapy with other agents or therapies. In addition, methods and assays for selecting appropriate agents that can modulate the availability of TTR and RBP in an individual are described herein.