Journal: eLife

Article Title: Contextual and cross-modality modulation of auditory cortical processing through pulvinar mediated suppression

doi: 10.7554/eLife.54157

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Article Snippet: Recombinant DNA reagent , AAV2/1-CB7-Cl-eGFP-WPRE-rBG , UPenn Vector Core , Addgene #105542 , .

Techniques: Recombinant, Plasmid Preparation, Staining, Software

Journal: JCI Insight

Article Title: AAV-mediated delivery of secreted acid α -glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice

doi: 10.1172/jci.insight.170199

Figure Lengend Snippet: ( A ) Experimental design: 3-month-old KO mice received a single injection of systemic vector (SYS; n = 5) at a dose of 2.5 × 10 13 vg/kg. AAV9-based liver-directed vector encoding untagged secretable hGAA served as a control (liver secreted, LS; n = 6). The samples were collected 1 month after dosing. ( B ) Western blot analysis of whole liver lysates with anti-human GAA antibody. Graphs show the amount and activity of the liver-expressed IGF2-tagged and untagged hGAA. ( C ) Western blot of plasma from SYS- and LS-treated KO mice. Ponceau stain was used to confirm equal protein loading. The amount of circulating precursor protein is quantified relative to a nonspecific band present in all samples. The amount and activity of the secreted hGAA are higher in LS- compared with SYS-treated KO mice. Statistical significance was determined by 1-way ANOVA and unpaired 2-tailed Student’s t test. Graphs represent mean ± SD. * P < 0.05; ** P < 0.01; *** P < 0.001; **** P < 0.0001.

Article Snippet: In vivo efficacy of AAV9-mediated gene transfer was evaluated following a single intravenous administration of AAV9.CAG.BiP.vIGF2.hGAAco.rBG.KanR (systemic; SYS) vector (Amicus proprietary) into young (3–3.5 months of age) or old (8–9 months of age) Gaa -KO mice (referred to as KO), a model of Pompe disease ( ).

Techniques: Injection, Plasmid Preparation, Western Blot, Activity Assay, Staining