Promoter Dependence of Plasmid–Pluronics Targeted α Galactosidase A Expression in Skeletal Muscle of Fabry Mice

Anderson–Fabry disease is an X-linked lysosomal storage disorder (LSD) caused by α-galactosidase A (AGA) deficiency [1]. There is evidence that LSDs could be corrected by gene transfer and expression and enzyme secretion from a subset of cells or a specific tissue [2–4]. Such gene therapy has several advantages over an alternative enzyme replacement approach [5,6], as it offers long-term therapeutic effect, eliminates risks associated with repeated parenteral administrations, and is less expensive.