Journal: Molecular Therapy Advances
Article Title: Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system
doi: 10.1016/j.omta.2026.201681
Figure Lengend Snippet: In vivo characterization of the full-length CP040 promoter packaged in the AAVDJ capsid (A) 1 × 10 13 vg/kg of AAVDJ-CP040 -eGFP or vehicle (1x PBS) was injected into the temporal vein of wild-type FVB/NJ neonatal mice (postnatal day 1–2). Necropsies were performed on the mice at six weeks of age, and tissue samples were analyzed for vg content and eGFP transcript levels. (B) Graph presenting vg copy numbers in different organs—cortex, cerebellum, kidneys, spleen, quadriceps, spinal cord, liver and heart. (C) Graph showing the fold change in e GFP transcript levels demonstrating promoter strength in the same organs as in (B). Data represent mean ± SEM, N = 10 mice, ∗∗ p < 0.01, ∗∗∗ p < 0.001, ∗∗∗∗ p < 0.0001, ns, not significant by one-way ANOVA. (D) Representative IF images showing eGFP expression along with neuronal (NeuN), astrocyte (GFAP), and oligodendrocyte (olig2) markers. Scale bar = 100 uM (E) Cells found to be double-positive for eGFP and one of the other markers (NeuN, GFAP, and olig2) were quantified from the IF images captured. Data presented as mean ± SEM, N = 3 mice, ∗∗∗∗ p < 0.0001 by one-way ANOVA.
Article Snippet: Five-week-old WT FVB/NJ (stock# 001800) mice were ordered from Jackson Laboratory.
Techniques: In Vivo, Injection, Expressing