Figure 1.
AAV vector-mediated expression of α-gal A in patient's fibroblasts. (A) Fibroblasts from a Fabry patient was transduced with AAV.CAαGBE. α-Gal A activity was determined 3 days after transduction. 1, Nontreated normal fibroblasts; 2, nontreated Fabry fibroblasts; 3, Fabry fibroblasts transduced with 5 × 104 AAV.CAαGBE genomes; 4, Fabry fibroblasts transduced with 1 × 105 AAV.CAαGBE genomes. (B) Fabry fibroblasts were incubated with the conditioned medium (CD) of HeLa cell clone (H#13) secreting α-gal A for overnight. 1, Nontreated fibroblasts; 2, incubation with CD; 3, incubation with CD in the presence of 1 mM mannose-6 phosphate; 4, incubation with CD in the presence of 1 mM glucose-6-phosphate. Values are presented as means ± SD.