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.
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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)
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Chemical References |
- Concanavalin A
- Hexosaminidases
- Hexosaminidase A
- Hexosaminidase B
- beta-N-Acetylhexosaminidases
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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
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