title: Pharmacoperones: targeting therapeutics toward diseases caused by protein misfolding.
abstract: Pharmacoperones are hydrophobic molecule drugs that enter cells and serve as a molecular framework to cause misfolded mutant proteins to fold properly and adopt a stable conformation compatible with proper intracellular trafficking. Pharmacoperones have successfully been used experimentally to rescue function of some misfolded proteins (enzymes, receptors, channels) that lead to disease. Identification of pharmacoperones by high-throughput screens of drug libraries will likely provide new molecules that may be potentially useful to treat diseases caused by protein misfolding.
authors: Research Support Network, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán and Universidad Nacional Autónoma de México, México, D.F., Mexico.
authors: Alfredo
authors: A
authors: Ulloa-Aguirre
authors: Research Support Network, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán and Universidad Nacional Autónoma de México, México, D.F., Mexico.
authors: Teresa
authors: T
authors: Zariñán
authors: Department of Internal Medicine, Texas Tech University Health Science Center, Lubbock, Texas, USA.|Department of Cell Biology/Biochemistry, Texas Tech University Health Science Center, Lubbock, Texas, USA.
authors: P Michael
authors: PM
authors: Conn
mesh_terms: D000818:Animals; D015195:Drug Design; D057166:High-Throughput Screening Assays; D006801:Humans; D057927:Hydrophobic and Hydrophilic Interactions; D018832:Molecular Chaperones / Q000737:chemistry / Q000378:metabolism*; D050505:Mutant Proteins / Q000737:chemistry / Q000378:metabolism; D017510:Protein Folding; D011506:Proteins / Q000737:chemistry / Q000378:metabolism*; D057165:Proteostasis Deficiencies / Q000188:drug therapy* / Q000473:pathology
chemical_list: D018832:Molecular Chaperones; D050505:Mutant Proteins; D011506:Proteins