caa a22 4500 469044209 CHVBK 20180323132819.0 cr unu---uuuuu 170328e19921101xx s 000 0 eng 10.1007/BF01800230 doi (NATIONALLICENCE)springer-10.1007/BF01800230 Long-term effects of bone marrow transplantation on lysosomal enzyme replacement in β-glucuronidase-deficient mice [Elektronische Daten] [G. Bou-Gharios, G. Adams, P. Pace, I. Olsen] Summary: This study uses bone marrow transplantation (BMT) between congenic strains of mice as an experimental model to examine enzyme replacement therapy of lysosomal storage diseases. Bone marrow cells from donor mice which have normal levels of the lysosomal enzyme β-glucuronidase (Gus), which is heat-stable, rapidly repopulated the haematopoietic compartment of irradiated recipient mice which have only low levels of a thermolabile form of this enzyme. Gus activity was found to increase progressively in the tissues of the recipients, including the liver, heart and skeletal muscle. Elevated levels were also observed in the kidney and brain. The increase in enzyme activity in the host tissues was not due to the presence of contaminating blood cells, but rather to the acquisition of new, heat-stable enzyme from the donor bone marrow cells. High levels of Gus activity persisted for at least 72 weeks, showing the potential therapeutic value of BMT for enzyme deficiency diseases. SSIEM and Kluwer Academic Publishers, 1992 Bou-Gharios G. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut Adams G. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut Pace P. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut Olsen I. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/6(1992-11-01), 899-910 0141-8955 15:6<899 1992 15 10545 https://doi.org/10.1007/BF01800230 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/BF01800230 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Bou-Gharios G. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut NATIONALLICENCE 700 1- Adams G. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut NATIONALLICENCE 700 1- Pace P. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut NATIONALLICENCE 700 1- Olsen I. Cell Enzymology Unit, Kennedy Institute of Rheumatology, Bute Gardens, W6 7DW, London, UK aut NATIONALLICENCE 773 0- Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/6(1992-11-01), 899-910 0141-8955 15:6<899 1992 15 10545 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer