BioTherapeutics Chemistry, Pfizer Worldwide Medicinal Chemistry, 200 CambridgePark Drive, Cambridge, MA 02140, USA.
Bioorg Med Chem Lett. 2013 Apr 1;23(7):1935-44. doi: 10.1016/j.bmcl.2013.01.089. Epub 2013 Feb 4.
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.
蛋白质错误折叠是一个新兴领域，跨越多个治疗领域，导致许多严重疾病。随着蛋白质错误折叠的生物途径变得更加清晰，该领域的小分子方法越来越受到关注。本文将综述文献中已知可调节错误折叠、稳定或蛋白质平衡的现有小分子。具体来说，将讨论以下靶点和方法：CFTR、葡萄糖脑苷脂酶、毒性寡聚物的调节、血清淀粉样蛋白 P (SAP) 片段和 HSF1 激活剂。
