A transgenic mouse line developed to express human amyloidogenic transthyretin cDNA in the brain.

To elucidate the role of amyloid deposits in the pathogenesis of degenerative disorders of brain, we attempted to develop transgenic mice producing amyloidogenic human variant transthyretin (TTR) in the brain. A minigene, in which the promoter region of the mouse myelin basic protein (MBP) gene was ligated to the human mutant TTR cDNA, was used for microinjection. A transgenic mouse line expressing the human TTR mRNA specifically in the brain was thus derived. However, human TTR was not detected in the brain or in the serum of these mice. These data suggest that the human mutant TTR mRNA is not efficiently translated in the mouse brain.