Gene targeting strategy for B-hTTR*V30M mice. The genome of mouse Ttr gene including 5’UTR and 3’UTR was replaced by human TTR genome including 5’UTR and 3’UTR in B-hTTR*V30M mice.

Strain specific analysis of TTR*V30M mRNA expression in wild-type C57BL/6 mice and B-hTTR*V30M mice by RT-PCR. Liver RNA was isolated from wildtype C57BL/6 mice (+/+) and heterozygous B-hTTR*V30M mice (H/+), then cDNA libraries were synthesized by reverse transcription, followed by PCR with mouse or human TTR primers. Mouse Ttr mRNA was detectable in wild-type C57BL/6 mice and heterozygous B-hTTR*V30M mice. Human TTR mRNA was detectable only in heterozygous B-hTTR*V30M mice but not in wild-type mice.

Strain specific TTR expression analysis in homozygous B-hTTR*V30M mice by ELISA. Plasma were isolated from wild-type C57BL/6 mice (+/+) and homozygous B-hTTR*V30M mice (H/H) and analyzed by ELISA with species-specific TTR ELISA kit. Mouse TTR was detectable in wild-type mice (A). Human TTR was exclusively detectable in homozygous B-hTTR*V30M mice (B).