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Enzyme replacement using liposome carriers in feline GM1 gangliosidosis fibroblasts

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Abstract

CURRENT emphasis in research on lysosomal storage diseases is directed towards enzyme replacement therapy (ERT) for these inherited enzyme deficiencies1–3. The rationale for ERT is based on the assumption that endocytosed enzyme would enter lysosomes and function in the degradation of accumulated substrates. However, inactivation of enzyme, immunological reactions and low cellular uptake have prevented successful application of this promising approach to therapy. Circumvention of these limitations requires the use of carriers which protect and stabilise replacement enzyme while enhancing uptake and subcellular distribution2–5. We report here the use of lipid vesicles (liposomes)6–9 as carriers for replacement enzyme in feline GM1 gangliosidosis fibroblasts which have deficient β-galactosidase (β-gal) activity and lysosomal storage of glycopeptides9–11. Our results demonstrate increased β-gal activity and corresponding catabolism of stored glycopeptides in feline GM1 gangliosidosis fibroblasts treated with liposomes containing β-gal.

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Authors and Affiliations

  1. Department of Comparative Medicine, University of Alabama in Birmingham, University Station, Birmingham, Alabama, 35294

    G. D. REYNOLDS, H. J. BAKER & R. H. REYNOLDS

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  1. G. D. REYNOLDS
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  2. H. J. BAKER
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  3. R. H. REYNOLDS
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REYNOLDS, G., BAKER, H. & REYNOLDS, R. Enzyme replacement using liposome carriers in feline GM1 gangliosidosis fibroblasts. Nature 275, 754–755 (1978). https://doi.org/10.1038/275754a0

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