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Fig. 5 | Orphanet Journal of Rare Diseases

Fig. 5

From: Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease

Fig. 5

Levels of Gb3 and Lyso-Gb3 in plasma, urine, and organs of FD mice treated with different doses of AAV2/8-hGLA. FD mice were intravenously administered formulation buffer or AAV2/8-hGLA at doses of 0.75E + 12, 1E + 12, 2.5E + 12, and 5E + 12 vg/kg, and tissues were collected at 12 weeks post-injection. Efficacy of different doses of AAV2/8-hGLA administration in clearing storage substrates Gb3 and Lyso-Gb3 in different tissues of FD mice. Data are presented as the mean ± SEM (n = 3–7). Data were analyzed using one-way ANOVA followed by Tukey’s multiple comparison tests. ****P < 0.0001, comparison of different doses of AAV2/8-hGLA treatment and FD group

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172