Fig. 3: Bioactivity of rAAVrh8-HexA and rAAVrh8-HexB in CNS and systemically.
From: Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial
a, HexA enzyme activity in CSF collected at the indicated time points. Analysis for HexA enzyme was only interpretable in patients with TSD. b, Total serum Hex levels collected at the indicated time points. The dashed line indicates normal Hex levels. c, C20:0 GM2 quantification by mass spectrometry. We note that juvenile patients have lower levels of GM2 as predicted by their disease subtype. d, CSF HexA activity levels from a generally correlated with GM2 20:0 levels.
