Journal of Biomedical Science is supported by the National Science and Technology Council (NSTC), Taiwan.
Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer
Enzyme replacement therapy (ERT) with α-galactosidase A (α-Gal A) is currently the most effective therapeutic strategy for patients with Fabry disease, a lysosomal storage disease. However, ERT has limitations...