ARTICLE | Cover Story

Reaching the surface in CF

January 7, 2010 8:00 AM UTC

Researchers at The Scripps Research Institute have found that inhibiting histone deacetylase 7 may improve ion transport in the lung cells of patients with cystic fibrosis by helping to properly fold mutant CFTR protein.1 Although the precise mechanism is not yet known,Proteostasis Therapeutics collaborating with the researchers to develop screens, and the company hopes to develop small molecule stimulators of protein folding to treat CF.

CF is caused by hereditary defects in cystic fibrosis transmembrane conductance regulator (CFTR), an ion pump that helps keep the lung epithelium hydrated and free of bacterial infections. Patients who inherit two copies of the DF508 allele, which is responsible for two-thirds of CF cases, make amisfolded version of the protein that does not get transported to the cell surface and instead is flagged by the endoplasmic reticulum for degradation by the proteasome...