Fig. 2: Validation of R26^SAM in mice.

a Mouse serum TTR levels were evaluated in R26^SAM/3aTtr (n = 7), R26^SAM/+ (n = 4) and WT (n = 4) for 7 months with monthly bleeds starting at 3 months. ELISA was used to determine protein levels in each mouse and values were plotted as the mean per group +/− SD. Monthly p-values can be found in Supplemental Table 2. Two independent replicates were conducted. b RT-qPCR determination of Ttr expression levels per tissue in R26^SAM/3aTtr mice (n = 3) relative to R26^SAM/+ plotted as the mean per group +/− SD. p-values of each tissue can be found in Supplemental Table 2. c RT-qPCR analysis of Rs1 expression in R26^SAM/3aRs1 mouse tissues (n = 4 mice per tissue) plotted as the mean per group +/− SD. p-values of each tissue can be found in Supplemental Table 2. d RNA-seq characterization of R26^SAM/3aTtr mouse tissues versus the parental R26^SAM/+ line. The transcriptome of each mouse tissue from five mice were sequenced to determine the number of target-specific TPM. The target gene is noted with a blue star and the adjacent genes with red squares. Kidney: r = 0.990, Spleen: r = 0.990, Brain: r = 0.999. e Schematic of LacZ knockout approach. The Ttr mouse coding sequence was replaced in mESCs starting at ATG with the LacZ coding sequence (Ttr^LacZ/+). f Tissues harvested from R26^SAM/3aTtr; R26^SAM/+ mice (n = 3), R26^SAM/+; Ttr^LacZ/+ mice (n = 3) and R26^SAM/3aTtr;Ttr^LacZ/+ mice (n = 3) stained with X-gal. Statistics: Asterisks (*) indicates significance, and the number of asterisks (*) indicates the number of 0s after the decimal point. One-tailed, unpaired Student’s t-test for (a–c) and Pearson’s correlation for (d).