Target Information

Target General Infomation
Target ID
T84173
Former ID
TTDC00014
Target Name
Glucosylceramidase
Gene Name
GBA
Synonyms
Acid beta-glucosidase; Alglucerase; Beta-glucocerebrosidase; D-glucosyl-N-acylsphingosine glucohydrolase; Imiglucerase; GBA
Target Type
Successful
Disease Metabolic disease [ICD9: 270-279; ICD10: E70-E89]
Metabolic disorders [ICD9: 270-279; ICD10: E70-E89]
BioChemical Class
Glycosylases
Target Validation
T84173
UniProt ID
P04062
EC Number
EC 3.2.1.45
Sequence
MEFSSPSREECPKPLSRVSIMAGSLTGLLLLQAVSWASGARPCIPKSFGYSSVVCVCNAT
YCDSFDPPTFPALGTFSRYESTRSGRRMELSMGPIQANHTGTGLLLTLQPEQKFQKVKGF
GGAMTDAAALNILALSPPAQNLLLKSYFSEEGIGYNIIRVPMASCDFSIRTYTYADTPDD
FQLHNFSLPEEDTKLKIPLIHRALQLAQRPVSLLASPWTSPTWLKTNGAVNGKGSLKGQP
GDIYHQTWARYFVKFLDAYAEHKLQFWAVTAENEPSAGLLSGYPFQCLGFTPEHQRDFIA
RDLGPTLANSTHHNVRLLMLDDQRLLLPHWAKVVLTDPEAAKYVHGIAVHWYLDFLAPAK
ATLGETHRLFPNTMLFASEACVGSKFWEQSVRLGSWDRGMQYSHSIITNLLYHVVGWTDW
NLALNPEGGPNWVRNFVDSPIIVDITKDTFYKQPMFYHLGHFSKFIPEGSQRVGLVASQK
NDLDAVALMHPDGSAVVVVLNRSSKDVPLTIKDPAVGFLETISPGYSIHTYLWRRQ
Drugs and Mode of Action
Drug(s) Taliglucerase alfa Drug Info Approved Metabolic disorders [532210], [542468]
Velaglucerase alfa Drug Info Approved Metabolic disorders [531351], [542489]
Isofagomine tartrate Drug Info Phase 2 Metabolic disease [542434], [548215]
Inhibitor (2R,3S,4S,5R)-2-hexylpiperidine-3,4,5-triol Drug Info [530788]
(2R,3S,4S,5R)-2-nonylpiperidine-3,4,5-triol Drug Info [530788]
(2R,3S,4S,5R)-2-propylpiperidine-3,4,5-triol Drug Info [530788]
1,5-Dideoxy-1,5-imino-D-xylitol Drug Info [529573]
2-(Acetylamino)-2-Deoxy-a-D-Glucopyranose Drug Info [551393]
Isofagomine tartrate Drug Info [536895], [536909]
L-Isofagomine Drug Info [529573]
N-(Propylamide-acetophenone)-1-deoxynojirimycin Drug Info [530532]
N-(Propylamide-benzophenone)-1-deoxynojirimycin Drug Info [530532]
Modulator Taliglucerase alfa Drug Info [532210]
Velaglucerase alfa Drug Info [531351]
Pathways
KEGG Pathway Other glycan degradation
Sphingolipid metabolism
Metabolic pathways
Lysosome
PathWhiz Pathway Sphingolipid Metabolism
Reactome Glycosphingolipid metabolism
WikiPathways Sphingolipid metabolism
References
Ref 531351Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5.
Ref 532210Nat Rev Drug Discov. 2013 Feb;12(2):87-90.
Ref 542434(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 542468(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 542489(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 548215Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800023168)
Ref 529573Bioorg 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 530532Bioorg 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.
Ref 530788Bioorg 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 531351Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5.
Ref 532210Nat Rev Drug Discov. 2013 Feb;12(2):87-90.
Ref 536895Isofagomine induced stabilization of glucocerebrosidase. Chembiochem. 2008 Nov 3;9(16):2643-9.
Ref 536909Identification 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 551393How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6.

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