Target Information
| Target General Infomation | |||||
|---|---|---|---|---|---|
| Target ID |
T84173
|
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| Former ID |
TTDC00014
|
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| Target Name |
Glucosylceramidase
|
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| Gene Name |
GBA
|
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| Synonyms |
Acid beta-glucosidase; Alglucerase; Beta-glucocerebrosidase; D-glucosyl-N-acylsphingosine glucohydrolase; Imiglucerase; GBA
|
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| Target Type |
Successful
|
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| Disease | Metabolic disorders [ICD9: 270-279; ICD10: E70-E89] | ||||
| Metabolic disease [ICD9: 270-279; ICD10: E70-E89] | |||||
| BioChemical Class |
Glycosylases
|
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| Target Validation |
T84173
|
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| UniProt ID | |||||
| EC Number |
EC 3.2.1.45
|
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| Sequence |
MEFSSPSREECPKPLSRVSIMAGSLTGLLLLQAVSWASGARPCIPKSFGYSSVVCVCNAT
YCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANHTGTGLLLTLQPEQKFQKVKGF GGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIRVPMASCDFSIRTYTYADTPDD FQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWTSPTWLKTNGAVNGKGSLKGQP GDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGLLSGYPFQCLGFTPEHQRDFIA RDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPEAAKYVHGIAVHWYLDFLAPAK ATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRGMQYSHSIITNLLYHVVGWTDW NLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHLGHFSKFIPEGSQRVGLVASQK NDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFLETISPGYSIHTYLWRRQ |
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| Drugs and Mode of Action | |||||
| Drug(s) | Taliglucerase alfa | Drug Info | Approved | Metabolic disorders | [1], [2] |
| Velaglucerase alfa | Drug Info | Approved | Metabolic disorders | [3], [4] | |
| Isofagomine tartrate | Drug Info | Phase 2 | Metabolic disease | [5], [6] | |
| Inhibitor | (2R,3S,4S,5R)-2-hexylpiperidine-3,4,5-triol | Drug Info | [7] | ||
| (2R,3S,4S,5R)-2-nonylpiperidine-3,4,5-triol | Drug Info | [7] | |||
| (2R,3S,4S,5R)-2-propylpiperidine-3,4,5-triol | Drug Info | [7] | |||
| 1,5-Dideoxy-1,5-imino-D-xylitol | Drug Info | [8] | |||
| 2-(Acetylamino)-2-Deoxy-a-D-Glucopyranose | Drug Info | [9] | |||
| Isofagomine tartrate | Drug Info | [10], [11] | |||
| L-Isofagomine | Drug Info | [8] | |||
| N-(Propylamide-acetophenone)-1-deoxynojirimycin | Drug Info | [12] | |||
| N-(Propylamide-benzophenone)-1-deoxynojirimycin | Drug Info | [12] | |||
| Modulator | Taliglucerase alfa | Drug Info | [1] | ||
| Velaglucerase alfa | Drug Info | [3] | |||
| Pathways | |||||
| KEGG Pathway | Other glycan degradation | ||||
| Sphingolipid metabolism | |||||
| Metabolic pathways | |||||
| Lysosome | |||||
| PathWhiz Pathway | Sphingolipid Metabolism | ||||
| Reactome | Glycosphingolipid metabolism | ||||
| WikiPathways | Sphingolipid metabolism | ||||
| References | |||||
| REF 1 | Nat Rev Drug Discov. 2013 Feb;12(2):87-90. | ||||
| REF 2 | (http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7444). | ||||
| REF 3 | Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5. | ||||
| REF 4 | (http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7464). | ||||
| REF 5 | (http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7410). | ||||
| REF 6 | Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800023168) | ||||
| REF 7 | Bioorg Med Chem. 2010 Apr 1;18(7):2645-50. Epub 2010 Feb 20.Synthesis of new beta-1-C-alkylated imino-L-iditols: A comparative study of their activity as beta-glucocerebrosidase inhibitors. | ||||
| REF 8 | Bioorg Med Chem. 2008 Aug 1;16(15):7330-6. Epub 2008 Jun 18.In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures. | ||||
| REF 9 | How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. | ||||
| REF 10 | Isofagomine induced stabilization of glucocerebrosidase. Chembiochem. 2008 Nov 3;9(16):2643-9. | ||||
| REF 11 | Identification of pharmacological chaperones for Gaucher disease and characterization of their effects on beta-glucocerebrosidase by hydrogen/deuterium exchange mass spectrometry. Chembiochem. 2008 Nov 3;9(16):2650-62. | ||||
| REF 12 | Bioorg Med Chem. 2010 Jan 1;18(1):267-73. Epub 2009 Oct 31.Nanomolar affinity, iminosugar-based chemical probes for specific labeling of lysosomal glucocerebrosidase. | ||||
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