. Author manuscript; available in PMC: 2015 May 1.

Published in final edited form as: Pharmacol Res. 2014 Apr 18;0:3–9. doi: 10.1016/j.phrs.2014.04.002

Table 1.

Select pharmacological chaperones used to enhance proteostasis of lysosomal enzymes, GPCRs and ion channels. See Figure 4 for chemical structures.

Protein type	Deficient protein	Disease	Pharmacological chaperones
Lysosomal enzymes	α-galactosidase A	Fabry disease	DGJ (56)
Lysosomal enzymes	Acid β-glucosidase	Gaucher disease	NN-DNJ (63)

Ion channels	hERG channel	Long QT syndrome	E4031 (69)
	CFTR	Cystic fibrosis	RDR1 (78)
	nAChR	Many such as epilepsy	Nicotine (72, 73)
	GABA_A receptor	Epilepsy	GABA (71)

GPCRs	V₂ vasopressin receptor	Nephrogenic diabetes insipidus	SR121463 (86)
	GnRH receptor	Hypogonadotropic hypogonadsm	IN3 (90)
	Opsin	Retinitis pigmentosa	11-cis-7-ring-retinal (94)
	δ-opioid receptor	Pain	Naltrexone (97)