fig1

Figure 1. EV-GLA therapeutic effect. (A) In vivo (left) and ex vivo (right) fluorescence imaging (FLI) of Fabry KO mice treated with either intraarterial (i.a.) or intravenous (i.v.) administration of DiR-labeled EV-GLA (1 mg/kg of GLA) in comparison to the non-treated controls (Cntrl). (B) FLI signal quantification to compare the biodistribution of EV-GLA following i.v. or i.a. administration. (C) Confocal images of brain parenchyma showing DiR fluorescent signal of EV-GLA (red) and DAPI-labeled cell nuclei (blue). Scale bar represents 20 µm. (D) GLA enzymatic activity 1 h post-administration in brains of Fabry KO mice treated with GLA or EV-GLA via i.v. or i.a. administrations (E) Loss of Gb3 in KO mice that received an i.v. single dose of EV-GLA, as measured by LC-HRMS seven days after dosing (figure used with permission from Figure 5 of Seras-Franzoso et al. Available from: https://doi.org/10.1002/jev2.12058^[25]). FLI: Luorescence imaging; GLA: alpha-galactosidase A.