Acta Neuropathologica

, Volume 50, Issue 1, pp 9–17 | Cite as

Enzyme replacement treatment for Tay-Sachs disease brain cells in culture utilizing Concanavalin A-mediated hexosaminidase A uptake: Biochemical and morphological evidence of GM2 mobilization

  • Steven E. Brooks
  • Linda M. Hoffman
  • Masazumi Adachi
  • Daniel Amsterdam
  • Larry Schneck
Original Works

Summary

When Concanavalin A (Con A) is bound to the cell membrane, it functions as an artificial enzyme receptor, mediating the binding and intracellular incorporation of significant amounts of exogenous hexosaminidase A (Hex A) into Tay-Sachs disease (TSD) glial cells. The treated cells retained almost 50% of incorporated Hex A activity after 3 days incubation in Hex A free medium. Hex A was released from Con A within the cell and was available as free enzyme. Biochemical analysis of gangliosides in Con A and Hex A treated cells depicted a greater than 50% reduction in stored GM2 ganglioside and a fourfold reduction in GM2label (14C) when compared to controls. Ultrastructural evidence of GM2 breakdown is presented which supports the biochemical and labeling studies.

Key words

Enzyme replacement Hex A TSD Con A Brain cells GM2 

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Copyright information

© Springer-Verlag 1980

Authors and Affiliations

  • Steven E. Brooks
    • 1
    • 2
  • Linda M. Hoffman
    • 1
    • 2
  • Masazumi Adachi
    • 1
    • 2
  • Daniel Amsterdam
    • 1
    • 2
  • Larry Schneck
    • 1
    • 2
  1. 1.Neuroscience Center of the Kingsbrook Jewish Medical CenterBrooklynUSA
  2. 2.Downstate Medical Center of the State University of New YorkBrooklynUSA

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