Chemical chaperone
- Cellular stress due to impairment of collagen prolyl hydroxylation complex is rescued by the chaperone 4-phenylbutyrate
Editor's choice: Mutations in the collagen 3-prolyl hydroxylation complex cause a cellular stress that is rescued by the chaperone ability of 4-phenylbutyrate.
- Restoration of mutant bestrophin-1 expression, localisation and function in a polarised epithelial cell model
Summary: Chemical chaperone 4PBA fully restores Cl− conductance activity for mutant bestrophin-1 proteins associated with inherited retinal dystrophy, autosomal recessive bestrophinopathy.