α-Galactosidase delivery using 30Kc19-human serum albumin nanoparticles for effective treatment of Fabry disease

Hong Jai Lee, Hee Ho Park, Youngsoo Sohn, Jina Ryu, Ju Hyun Park, Won Jong Rhee, Tai Hyun Park

Research output: Contribution to journalArticlepeer-review

24 Scopus citations

Abstract

Fabry disease is a genetic lysosomal storage disease caused by deficiency of α-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 α-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 α-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 α-galactosidase stability. 30Kc19-HSA nanoparticles loaded with the α-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 α-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.

Original languageEnglish
Pages (from-to)10395-10402
Number of pages8
JournalApplied Microbiology and Biotechnology
Volume100
Issue number24
DOIs
StatePublished - 1 Dec 2016

Bibliographical note

Publisher Copyright:
© 2016, Springer-Verlag Berlin Heidelberg.

Keywords

  • Drug delivery
  • Enzyme replacement therapy
  • Enzyme stability
  • Fabry disease
  • Protein nanoparticle

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