Treatment of Gaucher disease with an enzyme inhibitor
dc.contributor.author | Radin, Norman S. | en_US |
dc.date.accessioned | 2006-09-11T16:21:13Z | |
dc.date.available | 2006-09-11T16:21:13Z | |
dc.date.issued | 1996-04 | en_US |
dc.identifier.citation | Radin, Norman S.; (1996). "Treatment of Gaucher disease with an enzyme inhibitor." Glycoconjugate Journal 13(2): 153-157. <http://hdl.handle.net/2027.42/45689> | en_US |
dc.identifier.issn | 0282-0080 | en_US |
dc.identifier.issn | 1573-4986 | en_US |
dc.identifier.uri | https://hdl.handle.net/2027.42/45689 | |
dc.identifier.uri | http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=retrieve&db=pubmed&list_uids=8737239&dopt=citation | en_US |
dc.description.abstract | The hypothesis is offered predicting that Caucher patients could be treated with a drug that slows the synthesis of glucosylceramide, the lipid that accumulates in this disorder. The present therapeutic approach involves augmenting the defective enzyme, glucosylceramide β-glucosidase, with exogenous β-glucosidase isolated from human tissue. This spectacularly expensive mode of treatment should be replaceable with a suitable enzyme inhibitor that simply slows formation of the lipid and matches the rate of synthesis with the rate of the defective, slowly working β-glucosidase. Several drugs that possess this ability are available, the best known of which is 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), a designer inhibitor that resembles the synthase's substrate and product. PDMP has been found to be effective in mice, rats, fish, and a wide variety of cultured cells. Its use, at suitable dosages, seems to be harmless, although long-term tests have not been made. The lack of suitable animal models of Gaucher disease has made it difficult to test the hypothesis adequately, but PDMP does rapidly lower the levels of glucosylceramide in normal animal tissues and the animals evidently do well with the lowered levels of glucosylceramide and its more complex glycolipid metabolites. | en_US |
dc.format.extent | 591701 bytes | |
dc.format.extent | 3115 bytes | |
dc.format.mimetype | application/pdf | |
dc.format.mimetype | text/plain | |
dc.language.iso | en_US | |
dc.publisher | Kluwer Academic Publishers; Chapman & Hall ; Springer Science+Business Media | en_US |
dc.subject.other | Life Sciences | en_US |
dc.subject.other | Pathology | en_US |
dc.subject.other | Biochemistry, General | en_US |
dc.subject.other | Glucosylceramidase Insufficiency | en_US |
dc.subject.other | Gaucher Disease Chemotherapy | en_US |
dc.subject.other | Glucosylceramide Synthease Inhibition | en_US |
dc.subject.other | PDMP (1-phenyl-2-decanoylamino-3-morpholino-1-propanol) | en_US |
dc.title | Treatment of Gaucher disease with an enzyme inhibitor | en_US |
dc.type | Article | en_US |
dc.subject.hlbsecondlevel | Biological Chemistry | en_US |
dc.subject.hlbsecondlevel | Dentistry | en_US |
dc.subject.hlbsecondlevel | Chemical Engineering | en_US |
dc.subject.hlbsecondlevel | Chemistry | en_US |
dc.subject.hlbsecondlevel | Public Health | en_US |
dc.subject.hlbtoplevel | Engineering | en_US |
dc.subject.hlbtoplevel | Science | en_US |
dc.subject.hlbtoplevel | Health Sciences | en_US |
dc.description.peerreviewed | Peer Reviewed | en_US |
dc.contributor.affiliationum | Mental Health Research Institute and Division of Nephrology MSRBII, University of Michigan, 1550 W. Medical Centre Drive, 48109-0676, Ann Arbor, MI, USA | en_US |
dc.contributor.affiliationumcampus | Ann Arbor | en_US |
dc.identifier.pmid | 8737239 | en_US |
dc.description.bitstreamurl | http://deepblue.lib.umich.edu/bitstream/2027.42/45689/1/10719_2004_Article_BF00731489.pdf | en_US |
dc.identifier.doi | http://dx.doi.org/10.1007/BF00731489 | en_US |
dc.identifier.source | Glycoconjugate Journal | en_US |
dc.owningcollname | Interdisciplinary and Peer-Reviewed |
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