Table 1.
F508del-CFTR corrector compounds show distinct profiles of correction for other ER-retained proteins
| Corrector | CFTR F508del | hERG G601S | hERG F805C | SUR1 A116P | SUR1 V187D | V2R L292P | V2R V206D |
|---|---|---|---|---|---|---|---|
| VRT-325 |
+ |
+ |
- |
- |
ND |
- |
ND |
| Glycerol |
+ |
ND |
ND |
+ |
ND |
ND |
+/− |
| 29°C |
++ |
+ |
+ |
+ |
+ |
++ |
++ |
| KM60 |
+ |
+ |
- |
+ |
+ |
- |
+ |
| KM57 |
+/− |
++ |
- |
+/− |
- |
- |
+/− |
| ABT-888 |
+ |
- |
ND |
+ |
+ |
- |
+ |
| Glafenine |
+ |
+ |
- |
+ |
- |
- |
- |
| RDR1 |
+ |
- |
- |
+ |
+ |
- |
- |
| Ouabain |
+ |
+/−* |
+* |
+ |
+ |
+ |
+ |
| Carbamazepine |
+ |
- |
ND |
- |
+/− |
- |
ND |
| Latonduine |
+ |
+/− |
- |
+ |
++ |
+/− |
+ |
| Astemizole |
ND |
+ |
- |
ND |
ND |
ND |
ND |
| Glibenclamide | ND | ND | ND | ++ | ++ | ND | ND |
A qualitative assessment of correction as determined by glycosylation status in immunoblotting is shown for each mutation following treatment with a corrector compound, where “-“ indicates no correction observed, “+/−“ indicates slight correction, “+” and “++” indicate more and best correction observed, respectively, and “ND” indicates not determined. Asterisks indicate that an intermediate glycoform was observed, but no correction. Astemizole and glibenclamide were included as positive controls for correction of hERG G601S and SUR1 mutants, respectively.