General Information of the Protein
Protein ID
PT01904
Protein Name
Lysosomal alpha-glucosidase
Secondarily
Protein Name
Acid maltase
Aglucosidase alfa
Gene Name
GAA
Sequence
MGVRHPPCSHRLLAVCALVSLATAALLGHILLHDFLLVPRELSGSSPVLEETHPAHQQGASRPGPRDAQAHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGAQMGQPWCFFPPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLHFTIKDPANRRYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLFFADQFLQLSTSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLALEDGGSAHGVFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGYPFMPPYWGLGFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDGFRDFPAMVQELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKVWPGSTAFPDFTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELENPPYVPGVVGGTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISRSTFAGHGRYAGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVRWTQLGAFYPFMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAGETVARPLFLEFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPVEALGSLPPPPAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQPMALAVALTKGGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQLQKVTVLGVATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC
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Organism
Homo sapiens, Human
Protein Classification
Enzyme
>
Hydrolase
Function
Essential for the degradation of glycogen in lysosomes (PubMed:1856189, PubMed:7717400, PubMed:14695532, PubMed:18429042). Has highest activity on alpha-1,4-linked glycosidic linkages, but can also hydrolyze alpha-1,6-linked glucans (PubMed:29061980).
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Uniprot ID
Primary ID:
P10253

Secondarily ID:
Q09GN4
Q14351
Q16302
Q8IWE7
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Ensembl ID
ENSG00000171298
HGNC ID
HGNC:4065
Subcellular Location
Lysosome
Lysosome membrane
Map of Molecular Bioactivity Related to the Protein
Map of Molecular Bioactivity Related to the Protein

Protein
Cell Line
Compound

Bioactivity Value:

<= 0.1 μM
> 0.1 μM and <= 10 μM
> 10 μM
Imprecise Activity
Table of Molecular Bioactivities Related to the Protein
Cell Line ID: CL000034 , Caco-2
 Cell Line Info 
Compound ID Compound Name Compound Formula
CP0036230
 Compound Info 
(2R,3R,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol, 10
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C6H13NO4
  1
1
IC50 = 60 nM
 Bioactivity Info 
   TI
   LI
   LO
   TS
CP0053066
 Compound Info 
(1S,2S,3R,4S,5S)-5-(1,3-dihydroxypropan-2-ylamino)-1-(hydroxymethyl)cyclohexane-1,2,3,4-tetrol
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C10H21NO7
  1
1
IC50 = 70 nM
 Bioactivity Info 
   TI
   LI
   LO
   TS
CP0049663
 Compound Info 
2,6-Bis-hydroxymethyl-piperidine-3,4,5-triol
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C7H15NO5
  1
1
IC50 = 100 nM
 Bioactivity Info 
   TI
   LI
   LO
   TS
CP0053508
 Compound Info 
(2R,3R,4R,5S)-1-(2-hydroxyethyl)-2-(hydroxymethyl)piperidine-3,4,5-triol
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C8H17NO5
  1
1
IC50 = 1000 nM
 Bioactivity Info 
   TI
   LI
   LO
   TS
CP0055762
 Compound Info 
(2R,3R,4R,5R,6R)-5-((2R,3R,4R,5S,6R)-5-((2R,3R,4S,5S,6R)-3,4-dihydroxy-6-methyl-5-((1S,4R,5S,6S)-4,5,6-trihydroxy-3-(hydroxymethyl)cyclohex-2-enylamino)-tetrahydro-2H-pyran-2-yloxy)-3,4-dihydroxy-6-(hydroxymethyl)-tetrahydro-2H-pyran-2-yloxy)-6-(hydroxymethyl)-tetrahydro-2H-pyran-2,3,4-triol
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C25H43NO18
  1
1
IC50 = 5000 nM
 Bioactivity Info 
   TI
   LI
   LO
   TS
Biochemical Assays
Compound ID Compound Name Compound Formula
CP0053508
 Compound Info 
(2R,3R,4R,5S)-1-(2-hydroxyethyl)-2-(hydroxymethyl)piperidine-3,4,5-triol
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C8H17NO5
  2
1 IC50 = 350 nM
2 IC50 = 2000 nM
CP0036230
 Compound Info 
(2R,3R,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol, 10
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C6H13NO4
  6
1 IC50 = 1000 nM
2 IC50 = 1500 nM
3 IC50 = 91900 nM
4 Ki = 59 nM
5 Ki = 150 nM
6 Ki = 25000 nM
CP0049663
 Compound Info 
2,6-Bis-hydroxymethyl-piperidine-3,4,5-triol
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C7H15NO5
  1
1 IC50 = 1000 nM
CP0053066
 Compound Info 
(1S,2S,3R,4S,5S)-5-(1,3-dihydroxypropan-2-ylamino)-1-(hydroxymethyl)cyclohexane-1,2,3,4-tetrol
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C10H21NO7
  1
1 IC50 = 5600 nM
CP0055762
 Compound Info 
(2R,3R,4R,5R,6R)-5-((2R,3R,4R,5S,6R)-5-((2R,3R,4S,5S,6R)-3,4-dihydroxy-6-methyl-5-((1S,4R,5S,6S)-4,5,6-trihydroxy-3-(hydroxymethyl)cyclohex-2-enylamino)-tetrahydro-2H-pyran-2-yloxy)-3,4-dihydroxy-6-(hydroxymethyl)-tetrahydro-2H-pyran-2-yloxy)-6-(hydroxymethyl)-tetrahydro-2H-pyran-2,3,4-triol
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C25H43NO18
  1
1 IC50 = 42000 nM
Clinical Information about the Protein
Target 1 ( Lysosomal alpha-glucosidase (GAA) )
Target Type Successful Target
Disease 1 Target-related Disease  1
1 Pompe disease [ICD-11: 5C51.3]
Clinical Trial Drug(s) 1 Clinical Trial Drug  1
1 Deoxynojirimycin
 Drug Info 
Phase 3
Pompe disease