Target Information
| Target General Infomation | |||||
|---|---|---|---|---|---|
| Target ID |
T28265
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| Former ID |
TTDS00285
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| Target Name |
Alpha-1-antitrypsin
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| Gene Name |
SERPINA1
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| Synonyms |
Alpha-1 protease inhibitor; Alpha-1-antiproteinase; Alpha1-proteinase; PRO0684/PRO2209; SERPINA1
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| Target Type |
Successful
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| Disease | Alpha-1 antitrypsin deficiency [ICD9: 273.4; ICD10: E88.0] | ||||
| Chronic obstructive pulmonary disease; Cystic fibrosis [ICD9: 277.0, 460-519, 490-492, 494-496, 709.2; ICD10: E84, J00-J99, J40-J44, J47, L90.5] | |||||
| Emphysema [ICD9: 492; ICD10: J43] | |||||
| Unspecified [ICD code not available] | |||||
| Function |
Inhibitor of serine proteases. Its primary target is elastase, but it also has a moderate affinity for plasmin and thrombin.
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| BioChemical Class |
Serpin family
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| UniProt ID | |||||
| Sequence |
MPSSVSWGILLLAGLCCLVPVSLAEDPQGDAAQKTDTSHHDQDHPTFNKITPNLAEFAFS
LYRQLAHQSNSTNIFFSPVSIATAFAMLSLGTKADTHDEILEGLNFNLTEIPEAQIHEGF QELLRTLNQPDSQLQLTTGNGLFLSEGLKLVDKFLEDVKKLYHSEAFTVNFGDTEEAKKQ INDYVEKGTQGKIVDLVKELDRDTVFALVNYIFFKGKWERPFEVKDTEEEDFHVDQVTTV KVPMMKRLGMFNIQHCKKLSSWVLLMKYLGNATAIFFLPDEGKLQHLENELTHDIITKFL ENEDRRSASLHLPKLSITGTYDLKSVLGQLGITKVFSNGADLSGVTEEAPLKLSKAVHKA VLTIDEKGTEAAGAMFLEAIPMSIPPEVKFNKPFVFLMIEQNTKSPLFMGKVVNPTQK |
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| Drugs and Mode of Action | |||||
| Drug(s) | Glassia | Drug Info | Approved | Emphysema | [1], [2] |
| AGTC-0106 | Drug Info | Phase 2 | Alpha-1 antitrypsin deficiency | [3] | |
| Alphagen | Drug Info | Phase 1 | Chronic obstructive pulmonary disease; Cystic fibrosis | [4] | |
| Inhibitor | 2-Sulfhydryl-Ethanol | Drug Info | [5] | ||
| Glassia | Drug Info | [1] | |||
| Modulator | AGTC-0106 | Drug Info | [6] | ||
| alpha-1 proteinase inhibitor (inhaled), Alpha/Profile | Drug Info | ||||
| alpha-1 proteinase inhibitor, Hemosol | Drug Info | ||||
| Alphagen | Drug Info | [4] | |||
| Target Expression Profile (TEP) and Drug Resistance Mutation (DRM) | |||||
| TEP | EXP Info | ||||
| Pathways | |||||
| KEGG Pathway | Complement and coagulation cascades | ||||
| NetPath Pathway | IL6 Signaling Pathway | ||||
| PANTHER Pathway | Blood coagulation | ||||
| Pathway Interaction Database | p73 transcription factor network | ||||
| FOXA1 transcription factor network | |||||
| Reactome | Platelet degranulation | ||||
| COPII (Coat Protein 2) Mediated Vesicle Transport | |||||
| Cargo concentration in the ER | |||||
| WikiPathways | Complement and Coagulation Cascades | ||||
| NRF2 pathway | |||||
| Nuclear Receptors Meta-Pathway | |||||
| References | |||||
| REF 1 | Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5. | ||||
| REF 2 | (http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7487). | ||||
| REF 3 | ClinicalTrials.gov (NCT01054339) Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. U.S. National Institutes of Health. | ||||
| REF 4 | Emerging drugs for the treatment of chronic obstructive pulmonary disease. Expert Opin Emerg Drugs. 2006 May;11(2):275-91. | ||||
| REF 5 | DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Nucleic Acids Res. 2011 Jan;39(Database issue):D1035-4. Nucleic Acids Res. 2011 January | ||||
| REF 6 | First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9. doi: 10.1038/nbt0912-807. | ||||
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