Repository logo
  • English
  • 中文
Log In
Have you forgotten your password?
  1. Home
  2. College of Medicine / 醫學院
  3. School of Medicine / 醫學系
  4. 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
 
  • Details

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

Journal
European Journal of Medicinal Chemistry
Journal Volume
123
Pages
14-20
Date Issued
2016
Author(s)
Cheng W.-C.
Wang J.-H.
Li H.-Y.
Lu S.-J.
Hu J.-M.
Yun W.-Y.
Chiu C.-H.
Yang W.-B.
YIN-HSIU CHIEN  
WUH-LIANG HWU  
DOI
10.1016/j.ejmech.2016.07.025
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
https://scholars.lib.ntu.edu.tw/handle/123456789/525850
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
SDGs

[SDGs]SDG3

Other Subjects
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
Publisher
Elsevier Masson SAS
Type
journal article

臺大位居世界頂尖大學之列,為永久珍藏及向國際展現本校豐碩的研究成果及學術能量,圖書館整合機構典藏(NTUR)與學術庫(AH)不同功能平台,成為臺大學術典藏NTU scholars。期能整合研究能量、促進交流合作、保存學術產出、推廣研究成果。

To permanently archive and promote researcher profiles and scholarly works, Library integrates the services of “NTU Repository” with “Academic Hub” to form NTU Scholars.

總館學科館員 (Main Library)
醫學圖書館學科館員 (Medical Library)
社會科學院辜振甫紀念圖書館學科館員 (Social Sciences Library)

開放取用是從使用者角度提升資訊取用性的社會運動,應用在學術研究上是透過將研究著作公開供使用者自由取閱,以促進學術傳播及因應期刊訂購費用逐年攀升。同時可加速研究發展、提升研究影響力,NTU Scholars即為本校的開放取用典藏(OA Archive)平台。(點選深入了解OA)

  • 請確認所上傳的全文是原創的內容,若該文件包含部分內容的版權非匯入者所有,或由第三方贊助與合作完成,請確認該版權所有者及第三方同意提供此授權。
    Please represent that the submission is your original work, and that you have the right to grant the rights to upload.
  • 若欲上傳已出版的全文電子檔,可使用Open policy finder網站查詢,以確認出版單位之版權政策。
    Please use Open policy finder to find a summary of permissions that are normally given as part of each publisher's copyright transfer agreement.
  • 網站簡介 (Quickstart Guide)
  • 使用手冊 (Instruction Manual)
  • 線上預約服務 (Booking Service)
  • 方案一:臺灣大學計算機中心帳號登入
    (With C&INC Email Account)
  • 方案二:ORCID帳號登入 (With ORCID)
  • 方案一:定期更新ORCID者,以ID匯入 (Search for identifier (ORCID))
  • 方案二:自行建檔 (Default mode Submission)
  • 方案三:學科館員協助匯入 (Email worklist to subject librarians)

Built with DSpace-CRIS software - Extension maintained and optimized by 4Science