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Tissue specificity of rAAV2/8-HMBS-mediated HMB-synthase expression. Various tissues from saline- and rAAV2/8-HMBS-treated AIP mice were assayed for HMB-synthase activity (right) and vector copy number (left). The data presented are means + SD (n = 4). Note that vector copy number is shown on a logarithmic scale. AIP, acute intermittent porphyria.
