Fig. 1: Neuroimaging after BiT injection.
From: Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial
a, Immediate postoperation (postop) MRI depicting BiT injection of AAVrh8-HEXA and AAVrh8-HEXB (1,250 μl and 4.1 × 1013 vector genomes (vg) per thalamus). MRI without contrast showing T2-FLAIR hyperintensity in the thalamus at the injection site. The hyperintensity decreases over time (at 3 and 6 months). There is no evidence of hemorrhage or other major complication associated with the injection. b, Two patients demonstrated an increase in cortical T2 hypointensity, suggestive of myelin production, over the course of three months postinfusion. The effect was observed in the frontoparietal cortex, but not in the temporal cortex. c, DTI for five patients taken at baseline and 24 weeks after treatment showing neural tract fiber growth (green) and loss (red). d, Quantification of percent change in tract fiber numbers and volume from DTI data at 12 and 24 weeks posttreatment. DWI, diffusion-weighted imaging; F, female; mo, months, Pt., patient; SWI, susceptibility-weighted imaging.
