Target Information
| Target General Infomation | |||||
|---|---|---|---|---|---|
| Target ID |
T21678
|
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| Former ID |
TTDC00016
|
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| Target Name |
Alpha-galactosidase A
|
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| Gene Name |
GLA
|
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| Synonyms |
Agalsidase; Alpha-D-galactosidase A; Alpha-D-galactoside galactohydrolase; Melibiase; GLA
|
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| Target Type |
Clinical Trial
|
||||
| Disease | Fabry's disease [ICD9: 272.7; ICD10: E75.2] | ||||
| BioChemical Class |
Glycosylases
|
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| Target Validation |
T21678
|
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| UniProt ID | |||||
| EC Number |
EC 3.2.1.22
|
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| Sequence |
MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP
DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADIDDSWKSIK SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQEIG GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT MQMSLKDLL |
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| Drugs and Mode of Action | |||||
| Drug(s) | Migalastat | Drug Info | Preregistration | Fabry's disease | [1] |
| PRX-102 | Drug Info | Phase 1/2 | Fabry's disease | [2] | |
| Inhibitor | (+)-5-deoxyadeenophorine | Drug Info | [3] | ||
| 1-deoxygalactonojirimycin | Drug Info | [4] | |||
| 2,5-dideoxy-2,5-imino-D-altritol | Drug Info | [4] | |||
| Alpha-D-Mannose | Drug Info | [5] | |||
| Beta-1-C-butenyl-1-deoxygalactonojirimycin | Drug Info | [4] | |||
| Beta-1-C-Butyl-1-deoxygalactonojirimycin | Drug Info | [4] | |||
| CALYSTEGINE B2 | Drug Info | [6] | |||
| DEOXYGALACTONOJIRIMYCIN | Drug Info | [7] | |||
| Fucose | Drug Info | [5] | |||
| Modulator | Migalastat | Drug Info | [8], [9] | ||
| PRX-102 | Drug Info | [10] | |||
| Pathways | |||||
| KEGG Pathway | Galactose metabolism | ||||
| Glycerolipid metabolism | |||||
| Sphingolipid metabolism | |||||
| Glycosphingolipid biosynthesis - globo series | |||||
| Lysosome | |||||
| PathWhiz Pathway | Sphingolipid Metabolism | ||||
| Galactose Metabolism | |||||
| Reactome | Glycosphingolipid metabolism | ||||
| WikiPathways | Sphingolipid metabolism | ||||
| References | |||||
| REF 1 | Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800019652) | ||||
| REF 2 | ClinicalTrials.gov (NCT01981720) Extension Study of PRX-102 for 24 Months. U.S. National Institutes of Health. | ||||
| REF 3 | Bioorg Med Chem Lett. 2006 Jun 15;16(12):3262-7. Epub 2006 Apr 5.Flexible synthesis and biological evaluation of novel 5-deoxyadenophorine analogues. | ||||
| REF 4 | Bioorg Med Chem. 2010 Jun 1;18(11):3790-4. Epub 2010 Apr 21.2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease. | ||||
| REF 5 | How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. | ||||
| REF 6 | Identification of the glycosidase inhibitors swainsonine and calystegine B2 in Weir vine (Ipomoea sp. Q6 [aff. calobra]) and correlation with toxicity. J Nat Prod. 1995 Jun;58(6):878-86. | ||||
| REF 7 | Bioorg Med Chem Lett. 2005 Aug 1;15(15):3596-9.Glycosidase inhibition by 1-glycosyl-4-phenyl triazoles. | ||||
| REF 8 | AT-1001: a high-affinity alpha3beta4 nAChR ligand with novel nicotine-suppressive pharmacology. Br J Pharmacol. 2015 Apr;172(7):1834-45. | ||||
| REF 9 | The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines. J Inherit Metab Dis. 2009 Jun;32(3):424-40. Epub 2009 Apr 18. | ||||
| REF 10 | Characterization of a chemically modified plant cell culture expressed human alpha-Galactosidase-A enzyme for treatment of Fabry disease. Mol Genet Metab. 2015 Feb;114(2):259-67. | ||||
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