Long-term intracellular retention of hexosaminidase A by Tay-Sachs disease brain and lung cells in vitro.

Abstract

Enzyme-replacement treatment for metabolic storage disorders has been widely studied using model cell culture systems. This study determines the long-term fate of human hexosaminidase A supplied to Tay-Sachs disease brain and lung cells. Hex A retention studies showed that the incorporated Hex A is retained in undiminished quantity by TSD lung cells maintained in stationary culture for 14 days. Tay-Sachs disease brain cells similarly followed for 28 days in stationary culture showed an initial reduction in Hex A for 3 days, after which the Hex A level stabilized and remained relatively constant for the next 25 days. Hexosaminidase B isoenzyme was found to accumulate in both cell lines during extended cultivation, despite the observation that significant amounts were excreted into the extracellular environment. The demonstration of long-term intracellular retention of exogenously supplied therapeutic enzyme by the target cells offers additional evidence for the feasibility of an enzyme-replacement approach for study and treatment of lysosomal storage disorders.

• Authors: S E Brooks, L M Hoffman, D Amsterdam, M Adachi, L Schneck
• Journal: Journal of neuroscience research (J Neurosci Res) Vol. 6 Issue 3 Pg. 381-8 ( 1981) ISSN: 0360-4012 [Print] United States
• PMID: 6457913 (Publication Type: Comparative Study, Journal Article)

Chemical References

• Concanavalin A
• Hexosaminidases
• Hexosaminidase A
• Hexosaminidase B
• beta-N-Acetylhexosaminidases

Topics

• Brain (cytology, enzymology)
• Cells, Cultured
• Concanavalin A (pharmacology)
• Hexosaminidase A
• Hexosaminidase B
• Hexosaminidases (metabolism)
• Humans
• In Vitro Techniques
• Lung (cytology, enzymology)
• Tay-Sachs Disease (physiopathology)
• Time Factors
• beta-N-Acetylhexosaminidases