S-Space College of Engineering/Engineering Practice School (공과대학/대학원) Dept. of Chemical and Biological Engineering (화학생물공학부) Journal Papers (저널논문_화학생물공학부)
alpha-Galactosidase delivery using 30Kc19-human serum albumin nanoparticles for effective treatment of Fabry disease
- Lee, Hong Jai; Park, Hee Ho; Sohn, Youngsoo; Ryu, Jina; Park, Ju Hyun; Rhee, Won Jong; Park, Tai Hyun
- Issue Date
- APPLIED MICROBIOLOGY AND BIOTECHNOLOGY Vol.100 No.24, pp. 10395-10402
- Fabry disease; Enzyme replacement therapy; Protein nanoparticle; Drug delivery; Enzyme stability
- Fabry disease is a genetic lysosomal storage disease caused by deficiency of alpha-galactosidase, the enzyme-degrading neutral glycosphingolipid that is transported to lysosome. Glycosphingolipid accumulation by this disease causes multi-organ dysfunction and premature death of the patient. Currently, enzyme replacement therapy (ERT) using recombinant alpha-galactosidase is the only treatment available for Fabry disease. To maximize the efficacy of treatment, enhancement of cellular delivery and enzyme stability is a challenge in ERT using alpha-galactosidase. In this study, protein nanoparticles using human serum albumin (HSA) and 30Kc19 protein, originating from silkworm, were used to enhance the delivery and intracellular alpha-galactosidase stability. 30Kc19-HSA nanoparticles loaded with the alpha-galactosidase were formed by desolvation method. 30Kc19-HSA nanoparticles had a uniform spherical shape and were well dispersed in cell culture media. 30Kc19-HSA nanoparticles had negligible toxicity to human cells. The nanoparticles exhibited enhanced cellular uptake and intracellular stability of delivered alpha-galactosidase in human foreskin fibroblast. Additionally, they showed enhanced globotriaosylceramide degradation in Fabry patients' fibroblasts. It is expected that 30Kc19-HSA protein nanoparticles could be used as an effective tool for efficient delivery and enhanced stability of drugs.
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