Target Information

Target General Infomation
Target ID
T21678
Former ID
TTDC00016
Target Name
Alpha-galactosidase A
Gene Name
GLA
Synonyms
Agalsidase; Alpha-D-galactosidase A; Alpha-D-galactoside galactohydrolase; Melibiase; GLA
Target Type
Clinical Trial
Disease Fabry's disease [ICD9: 272.7; ICD10: E75.2]
BioChemical Class
Glycosylases
Target Validation
T21678
UniProt ID
P06280
EC Number
EC 3.2.1.22
Sequence
MQLRNPELHLGCALALRFLALVSWDIPGARALDNGLARTPTMGWLHWERFMCNLDCQEEP
DSCISEKLFMEMAELMVSEGWKDAGYEYLCIDDCWMAPQRDSEGRLQADPQRFPHGIRQL
ANYVHSKGLKLGIYADVGNKTCAGFPGSFGYYDIDAQTFADWGVDLLKFDGCYCDSLENL
ADGYKHMSLALNRTGRSIVYSCEWPLYMWPFQKPNYTEIRQYCNHWRNFADIDDSWKSIK
SILDWTSFNQERIVDVAGPGGWNDPDMLVIGNFGLSWNQQVTQMALWAIMAAPLFMSNDL
RHISPQAKALLQDKDVIAINQDPLGKQGYQLRQGDNFEVWERPLSGLAWAVAMINRQEIG
GPRSYTIAVASLGKGVACNPACFITQLLPVKRKLGFYEWTSRLRSHINPTGTVLLQLENT
MQMSLKDLL
Drugs and Mode of Action
Drug(s) Migalastat Drug Info Preregistration Fabry's disease [547820]
PRX-102 Drug Info Phase 1/2 Fabry's disease [524520]
Inhibitor (+)-5-deoxyadeenophorine Drug Info [528124]
1-deoxygalactonojirimycin Drug Info [530905]
2,5-dideoxy-2,5-imino-D-altritol Drug Info [530905]
Alpha-D-Mannose Drug Info [551393]
Beta-1-C-butenyl-1-deoxygalactonojirimycin Drug Info [530905]
Beta-1-C-Butyl-1-deoxygalactonojirimycin Drug Info [530905]
CALYSTEGINE B2 Drug Info [551364]
DEOXYGALACTONOJIRIMYCIN Drug Info [527609]
Fucose Drug Info [551393]
Modulator Migalastat Drug Info [533046], [537257]
PRX-102 Drug Info [532932]
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 524520ClinicalTrials.gov (NCT01981720) Extension Study of PRX-102 for 24 Months. U.S. National Institutes of Health.
Ref 547820Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800019652)
Ref 527609Bioorg Med Chem Lett. 2005 Aug 1;15(15):3596-9.Glycosidase inhibition by 1-glycosyl-4-phenyl triazoles.
Ref 528124Bioorg 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 530905Bioorg 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 532932Characterization 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.
Ref 533046AT-1001: a high-affinity alpha3beta4 nAChR ligand with novel nicotine-suppressive pharmacology. Br J Pharmacol. 2015 Apr;172(7):1834-45.
Ref 537257The 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 551364Identification 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 551393How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6.

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