Control of protein levels by nucleic-acid-based technologies has proven to be a useful
research tool but lacks the advantages of small molecules with respect to cell permeability …

Protein folding is a complex, error-prone process that often results in an irreparable protein
by-product. These by-products can be recognized by cellular quality control machineries …

Gaucher disease (GD), an autosomal recessive disease, is characterized by accumulation of
glucosylceramide mainly in cells of the reticuloendothelial system, due to mutations in the …

Cystic fibrosis is the most common life-threatening recessively inherited disease in
Caucasians. Due to early provision of care in specialized reference centers and more …

Increasing numbers of inherited diseases are found to result from mutations that lead to
misfolded proteins. In many cases, the changes in conformation are relatively modest and …

Small molecules are proposed as potential drugs for the treatment of lysosomal storage
disorders (LSDs) such as Fabry disease and Gaucher disease, which are caused by …

G protein-coupled receptors (GPCR) comprise the largest family of drug targets. This is not
surprising as many signaling systems rely on this class of receptor to convert external and …

The process of quality control in the endoplasmic reticulum involves a variety of mechanisms
which ensure that only correctly folded proteins enter the secretory pathway. Among these …

After synthesis, proteins are folded into their native conformations aided by molecular
chaperones. Dysfunction in folding caused by genetic mutations in numerous genes causes …

Background and purpose: TRPM4 and TRPM5 are calcium‐activated non‐selective cation
channels with almost identical characteristics. TRPM4 is detected in several tissues …