Target Information
| Target General Information | Top | |||||
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| Target ID |
T61339
(Former ID: TTDR00001)
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| Target Name |
Alpha-glucosidase (GLA)
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| Synonyms |
Maltase-glucoamylase; Maltase; Glucosidosucrase; Glucosidoinvertase; Glucoinvertase; Alpha-glucoside hydrolase; Alpha-glucopyranosidase; Alpha-D-glucoside glucohydrolase; Alpha-D-glucosidase; Alpha-1;4-glucosidase
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| Gene Name |
GAA; GANAB; GANC
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| Target Type |
Successful target
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[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Inborn carbohydrate metabolism error ICD-11: 5C51 | |||||
| Function |
Breaks down starch and disaccharides to glucose.
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| UniProt ID | ||||||
| Sequence |
MGVRHPPCSHRLLAVCALVSLATAALLGHILLHDFLLVPRELSGSSPVLEETHPAHQQGA
SRPGPRDAQAHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGA QMGQPWCFFPPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLH FTIKDPANRRYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLF FADQFLQLSTSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLA LEDGGSAHGVFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGY PFMPPYWGLGFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDG FRDFPAMVQELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKV WPGSTAFPDFTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELEN PPYVPGVVGGTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISR STFAGHGRYAGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVR WTQLGAFYPFMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAG ETVARPLFLEFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPV EALGSLPPPPAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQP MALAVALTKGGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQ LQKVTVLGVATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC Click to Show/Hide
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| Drugs and Modes of Action | Top | |||||
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| Approved Drug(s) | [+] 2 Approved Drugs | + | ||||
| 1 | Cipaglucosidase alfa | Drug Info | Approved | Pompe disease | [2] | |
| 2 | Cipaglucosidase alfa | Drug Info | Approved in EU | Glycogen storage disease type II | [3] | |
| Clinical Trial Drug(s) | [+] 3 Clinical Trial Drugs | + | ||||
| 1 | Anamorelin | Drug Info | Phase 3 | Carbohydrate metabolism disorder | [4] | |
| 2 | Deoxynojirimycin | Drug Info | Phase 3 | Pompe disease | [5] | |
| 3 | AAV2/8-LSPhGAA | Drug Info | Phase 1 | Pompe disease | [6] | |
| Mode of Action | [+] 3 Modes of Action | + | ||||
| Replacement | [+] 2 Replacement drugs | + | ||||
| 1 | Cipaglucosidase alfa | Drug Info | [7] | |||
| 2 | Cipaglucosidase alfa | Drug Info | [8] | |||
| Modulator | [+] 3 Modulator drugs | + | ||||
| 1 | Anamorelin | Drug Info | [9], [10] | |||
| 2 | JBP-1 | Drug Info | [13], [14] | |||
| 3 | POP-1 | Drug Info | [13], [14] | |||
| Inhibitor | [+] 8 Inhibitor drugs | + | ||||
| 1 | Deoxynojirimycin | Drug Info | [1] | |||
| 2 | Maltose | Drug Info | [11] | |||
| 3 | 4,6-Dideoxyglucose | Drug Info | [12] | |||
| 4 | 6-Deoxy-Alpha-D-Glucose | Drug Info | [12] | |||
| 5 | Beta-D-Glucose | Drug Info | [12] | |||
| 6 | Double Oxidized Cysteine | Drug Info | [12] | |||
| 7 | Nicotinamide-Adenine-Dinucleotide | Drug Info | [12] | |||
| 8 | Tendamistat | Drug Info | [15] | |||
| Drug Property Profile of Target | Top | |
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| (1) Molecular Weight (mw) based Drug Clustering | (2) Octanol/Water Partition Coefficient (xlogp) based Drug Clustering | |
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| (3) Hydrogen Bond Donor Count (hbonddonor) based Drug Clustering | (4) Hydrogen Bond Acceptor Count (hbondacc) based Drug Clustering | |
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| (5) Rotatable Bond Count (rotbonds) based Drug Clustering | (6) Topological Polar Surface Area (polararea) based Drug Clustering | |
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| "RO5" indicates the cutoff set by lipinski's rule of five; "D123AB" colored in GREEN denotes the no violation of any cutoff in lipinski's rule of five; "D123AB" colored in PURPLE refers to the violation of only one cutoff in lipinski's rule of five; "D123AB" colored in BLACK represents the violation of more than one cutoffs in lipinski's rule of five | ||
| Co-Targets | Top | |||||
|---|---|---|---|---|---|---|
| Co-Targets | ||||||
| Target Poor or Non Binders | Top | |||||
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| Target Poor or Non Binders | ||||||
| Target-Related Models and Studies | Top | |||||
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| Target Validation | ||||||
| References | Top | |||||
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| REF 1 | Nitrogen-in-the-ring pyranoses and furanoses: structural basis of inhibition of mammalian glycosidases. J Med Chem. 1994 Oct 28;37(22):3701-6. | |||||
| REF 2 | FDA Approved Drug Products from FDA Official Website. 2023. Application Number: 761204 | |||||
| REF 3 | Cipaglucosidase Alfa: First Approval. Drugs. 2023 Jun;83(8):739-745. | |||||
| REF 4 | ClinicalTrials.gov (NCT01395914) Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): An Extension Study (ROMANA 3). U.S. National Institutes of Health. | |||||
| REF 5 | The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase. Hum Mutat. 2009 Dec;30(12):1683-92. | |||||
| REF 6 | ClinicalTrials.gov (NCT03533673) AAV2/8-LSPhGAA in Late-Onset Pompe Disease. U.S. National Institutes of Health. | |||||
| REF 7 | Cipaglucosidase Alfa: First Approval. Drugs. 2023 Jun;83(8):739-745. | |||||
| REF 8 | FDA Approved Drug Products from FDA Official Website. 2023. Application Number: 761204 | |||||
| REF 9 | Absorption, elimination, and metabolism of CS-1036, a novel -amylase inhibitor in rats and monkeys, and the relationship between gastrointestinal distribution and suppression of glucose absorption.Drug Metab Dispos.2013 Apr;41(4):878-87. | |||||
| REF 10 | Alpha-amylase inhibitor, CS-1036 binds to serum amylase in a concentration-dependent and saturable manner.Drug Metab Dispos.2014 Mar;42(3):326-33. | |||||
| REF 11 | DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Nucleic Acids Res. 2011 Jan;39(Database issue):D1035-41. | |||||
| REF 12 | How many drug targets are there Nat Rev Drug Discov. 2006 Dec;5(12):993-6. | |||||
| REF 13 | US patent application no. 2012,0251,516, PHARMACEUTICAL COMPOSITION FOR TREATING CANCER COMPRISING TRYPSINOGEN AND/OR CHYMOTRYPSINOGEN AND AN ACTIVE AGENT SELECTED FROM A SELENIUM COMPOUND, A VANILLOID COMPOUND AND A CYTOPLASMIC GLYCOLYSIS REDUCTION AGENT. | |||||
| REF 14 | EP patent application no. 2490711, A pharmaceutical composition for treating cancer comprising trypsinogen and/or chymotrypsinogen and an active agent selected from a selenium compound, a vanilloid compound and a cytoplasmic glycolysis reduction agent). | |||||
| REF 15 | Influence of Specific Signal Peptide Mutations on the Expression and Secretion of the alpha -Amylase Inhibitor Tendamistat in Streptomyces lividans. J Biol Chem. 1996 Jun 21;271(25):15244-52. | |||||
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