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. 2023 May 8;8(9):e168688. doi: 10.1172/jci.insight.168688

Figure 3. ICV delivery of ST3GAL5 restores gangliosides production in St3gal5–/– mouse model.

Figure 3

(A) Schematic of ICV delivery of ubiquitous human ST3GAL5 cDNA Kozak ORF3 (KORF3) in St3gal5–/– mouse model. (B) ddPCR quantification of rAAV9 vector genome and human ST3GAL5 transgene in the brain, liver, and heart of rAAV9.CB.hST3GAL5-treated St3gal5–/– mice. Mouse endogenous St3gal5 mRNA was quantified from brain, liver, and heart of St3gal5+/+ mice. Data are reported as the mean ± SD of 7–10 animals/group. Statistical analysis was performed by 2-tailed t test. (C) Mass spectrometry quantification of GM3 (18:0), GM2 (18:0), and LacCer (18:0) from the brain of St3gal5+/+ and St3gal5–/– mice, with (+) or without (–) rAAV9.CB.hST3GAL5 treatment. Data are reported as the mean ± SD of 3 animals/group. Statistical analysis was performed by 1-way ANOVA, followed by Sidak’s multiple comparisons test. (D) Representative images of major brain gangliosides in cortex of St3gal5+/+ and St3gal5–/– mice, with (+) or without (–) rAAV9.CB.hST3GAL5 treatment. GD1a and GD1b are indicated by green; nuclei are counterstained in blue. Magnification, 63×. Scale bar: 3 µm. Quantification is shown in Supplemental Figure 2. *P < 0.05, **P < 0.01, ***P < 0.001, ****P < 0.0001.