FIGURE 1.

VX-809 slows cyst growth and improves renal function. Representative images of H&E-stained kidney sections from a cystic 5-month-old RC/RC control mouse or mouse treated with VX-809 every other day for 60 days beginning at 3 months of age (A). Cyst area, measured with ImageJ (provided by National Institutes of Health), is expressed as a percentage. Columns represent the mean ± SE of the % cyst area (B). Statistical analysis was performed using an unpaired t-test. **p < .01. (C) Kidney to body weight ratio was measured using wet weight. Dots = number of animals. Liver and kidney function tests. Blood samples were taken from control, treated animals, and serum was analyzed for kidney and liver function. Values measured were blood urea nitrogen (BUN) (D); aspartate aminotransferase (AST) (E); alanine transaminase (ALT) (F); and. Analyses were performed by spectrophotometric measurement using a Vet Ace clinical chemistry automated random-access analyzer (Alfa Wasserman Diagnostic Technologies). Values are means ± SEM. *p < .05. Statistical analysis was performed using an unpaired t-test. Dots = number of animals. CFTR, NHE3, ENaC, and PC2 Expression. Representative images of Western blotting for CFTR, NHE3, ENaC, and PC2 expression in normal (WT) mice, untreated RC/RC mice, and RC/RC mice treated with VX-809 (30 mg/kg) every other day for 60 days beginning at 3 months of age. Animals were necropsied at 5 months. (G&J). Summary data for CFTR, NHE3, ENaC, and PC2 expression, respectively. (H&I, K&L). HSP27 expression (M). Summary data for Hsp27 (N). Columns represent averages ± standard errors. *p < .05. **p < .01. Data is ratioed to the loading control, vinculin. Statistical analysis was performed using an unpaired t-test. Dots = number of animals