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Response of H3N2b to IV AAV9 gene therapy in a GM1 gangliosidosis patient. (a) H3N2b in urine. (b) H3N2b in plasma. (c) H3N2b in CSF. (d) Changes of CSF H3N2b and β-galactosidase activity. The red dash-dotted lines show the cutoffs for normal ranges as derived from ROC curve analysis.
