Fig. 3.

Schematic overview of current and future treatment approaches for Fabry disease. a Enzyme replacement therapy. b Migalastat-based chaperone treatment. c Lucerastat- and Venglustat-based substrate reduction therapy. d Lentiviral-based gene correction in haematopoietic stem cells. e Adenoviral-based transduction of liver cells. rhAGAL recombinant human α-galactosidase A, Gb3 globotriaosylceramide, GCS glucosylceramide synthase, ER endoplasmic reticulum, gDNA genomic DNA, mRNA messenger RNA