Recent developments in targeting protein misfolding diseases

BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

卷:23

Article

Denny, Rajiah Aldrin¹  Gavrin, Lori Krim¹  Saiah, Eddine¹ 

[1] Pfizer Worldwide Med Chem, BioTherapeut Chem, Cambridge, MA 02140 USA

关键词: Protein misfolding;    Chaperone;    Cystic fibrosis;    Gaucher disease;    Toxic oligomer;    Serum amyloid P (SAP);    Proteostasis;   

DOI  :  10.1016/j.bmcl.2013.01.089

来源: Elsevier

【 摘 要 】

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. (C) 2013 Elsevier Ltd. All rights reserved.

【 授权许可】

Free