Receptor-Mediated Endocytosis of α-Galactosidase A in Human Podocytes in Fabry Disease
Author Information
Author(s): Prabakaran Thaneas, Nielsen Rikke, Larsen Jakob V., Sørensen Søren S., Rasmussen Ulla Feldt-, Saleem Moin A., Petersen Claus M., Verroust Pierre J., Christensen Erik I.
Primary Institution: Aarhus University
Hypothesis
The study investigates the role of specific endocytic receptors in the uptake of α-galactosidase A by human podocytes in Fabry disease.
Conclusion
The study identifies megalin, sortilin, and M6PR as key receptors for the delivery of α-galactosidase A to lysosomes in human podocytes, suggesting new pathways for enzyme replacement therapy in Fabry disease.
Supporting Evidence
- The study shows that podocytes take up recombinant α-Gal A after infusion in Fabry patients.
- Three endocytic receptors were identified as important for α-Gal A accumulation in human podocytes.
- Sortilin was found to be a new receptor for α-Gal A, alongside megalin and M6PR.
Takeaway
This study found that certain proteins help deliver a medicine to kidney cells in patients with Fabry disease, which could improve treatment options.
Methodology
The study used biochemical and morphological approaches, including affinity chromatography and immunocytochemistry, to analyze human podocytes and renal tissue.
Statistical Information
P-Value
p<0.001
Statistical Significance
p<0.001
Digital Object Identifier (DOI)
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