Molecular and clinical characterization of AAV2/8‐TBG‐h

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Molecular and clinical characterization of AAV2/8‐TBG‐h Molecular and clinical characterization of AAV2/8‐TBG‐h.Ethe1HA‐treated mice. Molecular and clinical characterization of AAV2/8‐TBG‐h.Ethe1HA‐treated mice. A single i.c. injection of 4 × 1013 vg/kg was performed in each P21 animal.A.PCR analysis of AAV2/8 tissue distribution.B.Western‐blot analysis of tissue homogenates using an anti‐(α)‐ETHE1 antibody: the h.ETHE1HA protein is detected only in the liver of AAV‐treated animals as a band slightly slower than that corresponding to the endogenous mouse Ethe1 protein present in all wt (+/+) tissues. SDH‐A, the 70 kDa subunit of succinate dehydrogenase is used as a protein‐loading standard.C.Immunofluorescence of liver using an α‐HA antibody. The h.ETHE1HA protein is diffusely distributed in the liver of AAV‐treated animals (−/− AAV, right panel), whereas it is absent in naïve −/− animals (left panel). Scale bar: 50 µm.D.Western‐blot analysis of h.ETHE1HA protein in liver: the amount detected in three AAV‐treated mice (−/− AAV) is comparable to that of wt littermates (+/+), whereas the protein is absent in a sample from a naïve −/− animal.E.SDO activity in liver expressed in nmol O2/min/mg: the activity of AAV‐treated Ethe1−/− samples (−/− AAV; red bar; n = 3) is comparable to that of wt samples (+/+; blue bar; n = 2). Note that SDO activity is virtually absent in untreated Ethe1−/− samples (−/−; black bar; n = 3).F.Kaplan–Meier survival probability graph. Significance was assessed by log‐rank test. Grey: untreated Ethe1−/− mice (n = 15); green: Ethe1−/− mice treated with NAC for 10 days (n = 7); blue: Ethe1−/− treated with NAC ad libitum (n = 10); red: AAV‐treated Ethe1−/− mice (n = 20).G.Variation of body weights over time: for each point the difference between AAV‐treated Ethe1−/− mice (red) versus wt littermates (blue) was significant (p < 0.001).H.Activity cage test: no significant difference in spontaneous locomotor activity was measured between −/− AAV‐treated mice (n = 11) versus wt (+/+) littermates at 3–5 months of age. Ivano Di Meo et al. EMBO Mol Med. 2012;4:1008-1014 © as stated in the article, figure or figure legend