Target Information
Target General Infomation | |||||
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Target ID |
T31514
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Former ID |
TTDC00023
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Target Name |
Lysosomal alpha-glucosidase
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Gene Name |
GAA
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Synonyms |
Acid maltase; Aglucosidase alfa; GAA
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Target Type |
Clinical Trial
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Disease | Pompe's disease [ICD10: E74.02] | ||||
Function |
Essential for the degradation of glygogen to glucose in lysosomes.
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BioChemical Class |
Glycosylases
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Target Validation |
T31514
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UniProt ID | |||||
EC Number |
EC 3.2.1.20
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Sequence |
MGVRHPPCSHRLLAVCALVSLATAALLGHILLHDFLLVPRELSGSSPVLEETHPAHQQGA
SRPGPRDAQAHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGA QMGQPWCFFPPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLH FTIKDPANRRYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLF FADQFLQLSTSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLA LEDGGSAHGVFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGY PFMPPYWGLGFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDG FRDFPAMVQELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKV WPGSTAFPDFTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELEN PPYVPGVVGGTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISR STFAGHGRYAGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVR WTQLGAFYPFMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAG ETVARPLFLEFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPV EALGSLPPPPAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQP MALAVALTKGGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQ LQKVTVLGVATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC |
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Drugs and Mode of Action | |||||
Inhibitor | (-)-uniflorine A | Drug Info | [530453] | ||
(R)-2,6-Bis-hydroxymethyl-piperidine-3,4,5-triol | Drug Info | [534661] | |||
2,5-imino-2,5,6-trideoxy-D-manno-heptitol | Drug Info | [534633] | |||
7-O-b-D-Glucopyranosyl-a-homonojirimycin | Drug Info | [529573] | |||
Alpha-7-Deoxyhomonojirimycin | Drug Info | [526494] | |||
Alpha-Homonojirimycin | Drug Info | [529573] | |||
Deoxynojirimycin | Drug Info | [529573] | |||
N-adamantanemethyloxypentyl-1-deoxynojirimycin | Drug Info | [530471] | |||
SALACINOL | Drug Info | [529528] | |||
UNIFLORINE B | Drug Info | [530453] | |||
VALIOLAMINE | Drug Info | [529573] | |||
Modulator | BMN-701 | Drug Info | [524362], [551880] | ||
GZ402666 | Drug Info | [524362] | |||
Pathways | |||||
KEGG Pathway | Galactose metabolism | ||||
Starch and sucrose metabolism | |||||
Metabolic pathways | |||||
Lysosome | |||||
Pathway Interaction Database | Notch-mediated HES/HEY network | ||||
PathWhiz Pathway | Galactose Metabolism | ||||
References | |||||
Ref 521814 | ClinicalTrials.gov (NCT00306072) Effect of Different Doses of Salacinol Extract on Glycemic and Insulinemic Response in Patients With Type 2 Diabetes. U.S. National Institutes of Health. | ||||
Ref 524362 | ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health. | ||||
Ref 524362 | ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health. | ||||
Ref 526494 | J Nat Prod. 2002 Dec;65(12):1875-81.New polyhydroxylated pyrrolidine, piperidine, and pyrrolizidine alkaloids from Scilla sibirica. | ||||
Ref 529528 | J Nat Prod. 2008 Jun;71(6):981-4. Epub 2008 Jun 12.Alpha-glucosidase inhibitor from Kothala-himbutu (Salacia reticulata WIGHT). | ||||
Ref 529573 | 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 530471 | Bioorg Med Chem Lett. 2009 Dec 1;19(23):6600-3. Epub 2009 Oct 8.Synthesis and evaluation of D-gluco-pyranocyclopropyl amines as potential glucosidase inhibitors. | ||||
Ref 534633 | J Nat Prod. 1998 May;61(5):625-8.Nitrogen-containing furanose and pyranose analogues from Hyacinthus orientalis. |
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