Fig. 1: GLA transgene expression and characterization in cultured hepatocytes. | Gene Therapy

Fig. 1: GLA transgene expression and characterization in cultured hepatocytes.

From: Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

Fig. 1

Kinetics of α-Gal A secretion following FLT190 vector transduction in A Huh7 hepatocyte cell line and B human primary hepatocytes. C α-Gal A secretion from Huh7 cells engineered to overexpress α-Gal A. D Glycosylation analysis of α-Gal A produced from Huh7 cells following PNGase F digestion – representative Western blot analyses are shown. Agalsidase alfa was used as a comparator. Data are mean ± SD (n = 3). α-Gal A = alpha galactosidase A; AAV = adeno-associated virus; Huh7 = human hepatocyte cell line; MOI = multiplicity of infection; PNGase F = Peptide:N-Glycosidase F; SD = standard deviation; vg = vector genomes.

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