https://scholars.lib.ntu.edu.tw/handle/123456789/531363| DC Field | Value | Language |
|---|---|---|
| dc.contributor.author | Cheng W.-C. | en-US |
| dc.contributor.author | Wang J.-H. | en-US |
| dc.contributor.author | Li H.-Y. | en-US |
| dc.contributor.author | Lu S.-J. | en-US |
| dc.contributor.author | Hu J.-M. | en-US |
| dc.contributor.author | Yun W.-Y. | en-US |
| dc.contributor.author | Chiu C.-H. | en-US |
| dc.contributor.author | Yang W.-B. | en-US |
| dc.contributor.author | YIN-HSIU CHIEN | en-US |
| dc.contributor.author | Hwu W.-L. | en-US |
| dc.date.accessioned | 2020-12-24T06:16:35Z | - |
| dc.date.available | 2020-12-24T06:16:35Z | - |
| dc.date.issued | 2016 | - |
| dc.identifier.issn | 0223-5234 | - |
| dc.identifier.uri | https://www.scopus.com/inward/record.uri?eid=2-s2.0-84979643798&doi=10.1016%2fj.ejmech.2016.07.025&partnerID=40&md5=52876c24f6ccad10c80a92e4f6a68cc8 | - |
| dc.identifier.uri | https://scholars.lib.ntu.edu.tw/handle/123456789/531363 | - |
| dc.description.abstract | A unique molecular library consisting of all sixteen synthetic ADMDP (1-aminodeoxy-DMDP) stereoisomers has been prepared and evaluated for inhibitory activity against α-Gal A, and ability to impart thermal stabilization of this enzyme. The results of this testing led us to develop a novel pharmacological chaperone for the treatment of Fabry disease. 3-Epimer ADMDP was found to be an effective pharmacological chaperone, able to rescue α-Gal A activity in the lymphoblast of the N215S Fabry patient-derived cell line, without impairment of cellular β-galactosidase activity. When 3-epimer ADMDP was administered with rh-α-Gal A (enzyme replacement therapy) for the treatment of Fabry patient-derived cell lines, improvements in the efficacy of rh-α-Gal A was observed, which suggests this small molecule can also provide clinical benefit of enzyme replacement therapy in Fabry disease. ? 2016 | - |
| dc.publisher | Elsevier Masson SAS | - |
| dc.relation.ispartof | European Journal of Medicinal Chemistry | - |
| dc.subject.other | alpha galactosidase; beta galactosidase; glycosidase inhibitor; recombinant human alpha galactosidase; unclassified drug; 1-aminodeoxy-2,5-dideoxy-2,5-iminomannitol; alpha galactosidase; enzyme inhibitor; iminosugar; mannitol; molecular library; pyrrolidine derivative; Article; carbon nuclear magnetic resonance; dose response; drug potency; drug potentiation; drug screening; drug synthesis; enzyme activity; enzyme inhibition; enzyme replacement; epimer; Fabry disease; human; human cell; IC50; isomer; lymphoblast; lysosome; nucleophilicity; pH; proton nuclear magnetic resonance; stereoisomerism; analogs and derivatives; chemistry; drug effects; enzyme replacement; enzyme stability; Fabry disease; molecular library; pharmacology; procedures; stereoisomerism; synthesis; tumor cell line; alpha-Galactosidase; Cell Line, Tumor; Enzyme Inhibitors; Enzyme Replacement Therapy; Enzyme Stability; Fabry Disease; Humans; Imino Pyranoses; Imino Sugars; Mannitol; Pyrrolidines; Small Molecule Libraries; Stereoisomerism | - |
| dc.subject.other | [SDGs]SDG3 | - |
| dc.title | Bioevaluation of sixteen ADMDP stereoisomers toward alpha-galactosidase A: Development of a new pharmacological chaperone for the treatment of Fabry disease and potential enhancement of enzyme replacement therapy efficiency | en_US |
| dc.type | journal article | - |
| dc.identifier.doi | 10.1016/j.ejmech.2016.07.025 | - |
| dc.identifier.pmid | 27474919 | - |
| dc.identifier.scopus | 2-s2.0-84979643798 | - |
| dc.relation.pages | 14-20 | - |
| dc.relation.journalvolume | 123 | - |
| item.openairecristype | http://purl.org/coar/resource_type/c_6501 | - |
| item.fulltext | no fulltext | - |
| item.cerifentitytype | Publications | - |
| item.grantfulltext | none | - |
| item.openairetype | journal article | - |
| crisitem.author.dept | Pediatrics | - |
| crisitem.author.dept | Pediatrics-NTUH | - |
| crisitem.author.dept | Medical Genetics-NTUH | - |
| crisitem.author.orcid | 0000-0001-8802-5728 | - |
| crisitem.author.parentorg | College of Medicine | - |
| crisitem.author.parentorg | National Taiwan University Hospital | - |
| crisitem.author.parentorg | National Taiwan University Hospital | - |
| Appears in Collections: | 醫學系 | |
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