title: Cellular assays for drug discovery in genetic disorders of intracellular trafficking.
abstract: Intracellular membrane trafficking is essential for organelle biogenesis, structure, and function; the exchange of material between organelles; and communication between the cell and its external environment. Genetic disorders affecting intracellular trafficking can lead to a variety of human diseases, but specific therapies for these diseases are notably lacking. In this article, we focus on how current knowledge about genetic disorders that affect intracellular trafficking can be used to develop strategies for cell-based assays in order to identify drugs using high-content screening approaches.
authors.affiliation: Telethon Institute of Genetics and Medicine, 80131 Naples, Italy; email: dematteis@tigem.it.
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: De Matteis
authors.forename: Mariella
authors.initials: M
authors.lastname: Vicinanza
authors.forename: Rossella
authors.initials: R
authors.lastname: Venditti
authors.forename: Cathal
authors.initials: C
authors.lastname: Wilson
mesh_terms: D000818:Animals; D055808:Drug Discovery; D030342:Genetic Diseases, Inborn / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D007425:Intracellular Membranes / Q000378:metabolism; D021381:Protein Transport