Target Information

Target General Infomation
Target ID
T28265
Former ID
TTDS00285
Target Name
Alpha-1-antitrypsin
Gene Name
SERPINA1
Synonyms
Alpha-1 protease inhibitor; Alpha-1-antiproteinase; Alpha1-proteinase; PRO0684/PRO2209; SERPINA1
Target Type
Successful
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.
BioChemical Class
Serpin family
UniProt ID
P01009
Sequence
MPSSVSWGILLLAGLCCLVPVSLAEDPQGDAAQKTDTSHHDQDHPTFNKITPNLAEFAFS
LYRQLAHQSNSTNIFFSPVSIATAFAMLSLGTKADTHDEILEGLNFNLTEIPEAQIHEGF
QELLRTLNQPDSQLQLTTGNGLFLSEGLKLVDKFLEDVKKLYHSEAFTVNFGDTEEAKKQ
INDYVEKGTQGKIVDLVKELDRDTVFALVNYIFFKGKWERPFEVKDTEEEDFHVDQVTTV
KVPMMKRLGMFNIQHCKKLSSWVLLMKYLGNATAIFFLPDEGKLQHLENELTHDIITKFL
ENEDRRSASLHLPKLSITGTYDLKSVLGQLGITKVFSNGADLSGVTEEAPLKLSKAVHKA
VLTIDEKGTEAAGAMFLEAIPMSIPPEVKFNKPFVFLMIEQNTKSPLFMGKVVNPTQK
Drugs and Mode of Action
Drug(s) Glassia Drug Info Approved Emphysema [531351], [542510]
AGTC-0106 Drug Info Phase 2 Alpha-1 antitrypsin deficiency [522924]
Alphagen Drug Info Phase 1 Chronic obstructive pulmonary disease; Cystic fibrosis [536223]
Inhibitor 2-Sulfhydryl-Ethanol Drug Info [551391]
Glassia Drug Info [531351]
Modulator AGTC-0106 Drug Info [525389]
alpha-1 proteinase inhibitor (inhaled), Alpha/Profile Drug Info
alpha-1 proteinase inhibitor, Hemosol Drug Info
Alphagen Drug Info [536223]
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 522924ClinicalTrials.gov (NCT01054339) Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency. U.S. National Institutes of Health.
Ref 531351Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5.
Ref 536223Emerging drugs for the treatment of chronic obstructive pulmonary disease. Expert Opin Emerg Drugs. 2006 May;11(2):275-91.
Ref 542510(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 525389First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9. doi: 10.1038/nbt0912-807.
Ref 531351Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5.
Ref 536223Emerging drugs for the treatment of chronic obstructive pulmonary disease. Expert Opin Emerg Drugs. 2006 May;11(2):275-91.
Ref 551391DrugBank 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

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