Data files

We generated AAV(L):bPGRN (L = liver, b = brain-penetrant), a liver-targeting adenovirus (AAV) encoding a fusion protein (8D3:PGRN) consisting of a single chain fragment variable (scFv) antibody recognizing mouse TfR (transferrin receptor) fused to human PGRN (hPGRN). To study the effects of AAV(L):bPGRN, we treated 6-week old wildtype or GRN,TMEM106B double knock-out mice with either 1.3 x 10^13 vg/kg AAV or isotonic saline solution (0.9% NaCl) intravenously via tail vain injection. 9-10 weeks ...

We generated AAV(L):bPGRN (L = liver, b = brain-penetrant), a liver-targeting adenovirus (AAV) encoding a fusion protein (8D3:PGRN) consisting of a single chain fragment variable (scFv) antibody recognizing mouse TfR (transferrin receptor) fused to human PGRN (hPGRN). Grn knockout (KO) mice were treated with either an AAV driving expression of 8D3-linked progranulin (AAV(L)-8D3-PGRN) in the liver (n=5) or saline (n=5). As a control, wildtype (WT) animals treated with saline (n=6) were included.