title: Recent developments in targeting protein misfolding diseases.
abstract: Protein misfolding is an emerging field that crosses multiple therapeutic areas and causes many serious diseases. As the biological pathways of protein misfolding become more clearly elucidated, small molecule approaches in this arena are gaining increased attention. This manuscript will survey current small molecules from the literature that are known to modulate misfolding, stabilization or proteostasis. Specifically, the following targets and approaches will be discussed: CFTR, glucocerebrosidase, modulation of toxic oligomers, serum amyloid P (SAP) sections and HSF1 activators.
authors: BioTherapeutics Chemistry, Pfizer Worldwide Medicinal Chemistry, 200 CambridgePark Drive, Cambridge, MA 02140, USA.
authors: Rajiah Aldrin
authors: RA
authors: Denny
authors: Lori Krim
authors: LK
authors: Gavrin
authors: Eddine
authors: E
authors: Saiah
mesh_terms: D006801:Humans; D008958:Models, Molecular; D017510:Protein Folding / Q000187:drug effects; D057165:Proteostasis Deficiencies / Q000188:drug therapy* / Q000378:metabolism; D054852:Small Molecule Libraries / Q000737:chemistry / Q000627:therapeutic use*; D013816:Thermodynamics
chemical_list: D054852:Small Molecule Libraries