Target Information
| Target General Information | Top | |||||
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| Target ID |
T63155
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| Target Name |
Pyruvate kinase PKLR (PKLR)
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| Synonyms |
Pyruvate kinase 1; Pyruvate kinase isozymes L/R; R-type/L-type pyruvate kinase; Red cell/liver pyruvate kinase
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| Gene Name |
PKLR
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| Target Type |
Successful target
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[1] | ||||
| Disease | [+] 2 Target-related Diseases | + | ||||
| 1 | Haemolytic anemia [ICD-11: 3A20-3A2Z] | |||||
| 2 | Lysosomal disease [ICD-11: 5C56] | |||||
| Function |
Pyruvate kinase that catalyzes the conversion of phosphoenolpyruvate to pyruvate with the synthesis of ATP, and which plays a key role in glycolysis. {ECO:0000269|PubMed:11960989}.
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| BioChemical Class |
Kinase
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| UniProt ID | ||||||
| EC Number |
EC 2.7.1.40
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| Sequence |
MSIQENISSLQLRSWVSKSQRDLAKSILIGAPGGPAGYLRRASVAQLTQELGTAFFQQQQ
LPAAMADTFLEHLCLLDIDSEPVAARSTSIIATIGPASRSVERLKEMIKAGMNIARLNFS HGSHEYHAESIANVREAVESFAGSPLSYRPVAIALDTKGPEIRTGILQGGPESEVELVKG SQVLVTVDPAFRTRGNANTVWVDYPNIVRVVPVGGRIYIDDGLISLVVQKIGPEGLVTQV ENGGVLGSRKGVNLPGAQVDLPGLSEQDVRDLRFGVEHGVDIVFASFVRKASDVAAVRAA LGPEGHGIKIISKIENHEGVKRFDEILEVSDGIMVARGDLGIEIPAEKVFLAQKMMIGRC NLAGKPVVCATQMLESMITKPRPTRAETSDVANAVLDGADCIMLSGETAKGNFPVEAVKM QHAIAREAEAAVYHRQLFEELRRAAPLSRDPTEVTAIGAVEAAFKCCAAAIIVLTTTGRS AQLLSRYRPRAAVIAVTRSAQAARQVHLCRGVFPLLYREPPEAIWADDVDRRVQFGIESG KLRGFLRVGDLVIVVTGWRPGSGYTNIMRVLSIS Click to Show/Hide
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| Drugs and Modes of Action | Top | |||||
|---|---|---|---|---|---|---|
| Approved Drug(s) | [+] 2 Approved Drugs | + | ||||
| 1 | Mitapivat | Drug Info | Approved | Hereditary haemolytic anemia | [2] | |
| 2 | Olipudase alfa | Drug Info | Approved | Gaucher disease | [3] | |
| Clinical Trial Drug(s) | [+] 1 Clinical Trial Drugs | + | ||||
| 1 | RP-L301 | Drug Info | Phase 1 | Pyruvate kinase deficiency | [1] | |
| Mode of Action | [+] 1 Modes of Action | + | ||||
| Activator | [+] 2 Activator drugs | + | ||||
| 1 | Mitapivat | Drug Info | [2] | |||
| 2 | Olipudase alfa | Drug Info | [4] | |||
| References | Top | |||||
|---|---|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04105166) Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD. U.S.National Institutes of Health. | |||||
| REF 2 | FDA Approved Drug Products from FDA Official Website. 2022. Application Number: 216196. | |||||
| REF 3 | FDA Approved Drug Products from FDA Official Website. 2022. Application Number: 761261. | |||||
| REF 4 | FDA Approved Drug Products from FDA Official Website. 2022. Application Number: 761261 | |||||
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