Fig. 7: Correlating Plasma α-Gal A to Gb3.
From: Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Correlating plasma α-Gal A to Gb3 using a nonlinear fit regression model Marquardt method in SAS System. Percentage (%) of remaining storage relative to untreated controls, Sigmoid (4PL) Fit: X = Log α-Gal A, Y = Logged Gb3 analysis is shown. The R2 values are the following: plasma = 0.89 (A), for kidney = 0.72 (B), and for heart = 0.82 (C). The main purpose of the curve fitting was to predict plasma α-Gal A values for various levels of Gb3 reduction (e.g. 50%), exploring log(plasma Gb3), log(kidney Gb3) and log(heart Gb3) values as the y responses. α-Gal A prediction at 10%, 20%, 50% and 70% remaining storage are shown in tables. Statistical packages SAS and GraphPad Prism were used for the analysis. α-Gal A = alpha galactosidase A; Gb3 = globotriaosylceramide.
