Target Information
| Target General Information | Top | |||||
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| Target ID |
T83585
(Former ID: TTDI02221)
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| Target Name |
Wiskott-Aldrich syndrome protein (WAS)
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| Synonyms |
WASp; IMD2
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| Gene Name |
WAS
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| Target Type |
Clinical trial target
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[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Qualitative platelet defect [ICD-11: 3B62] | |||||
| Function |
Important for efficient actin polymerization. Possible regulator of lymphocyte and platelet function. Mediates actin filament reorganization and the formation of actin pedestals upon infection by pathogenic bacteria. In addition to its role in the cytoplasmic cytoskeleton, also promotes actin polymerization in the nucleus, thereby regulating gene transcription and repair of damaged DNA. Promotes homologous recombination (HR) repair in response to DNA damage by promoting nuclear actin polymerization, leading to drive motility of double-strand breaks (DSBs). Effector protein for Rho-type GTPases that regulates actin filament reorganization via its interaction with the Arp2/3 complex.
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| UniProt ID | ||||||
| Sequence |
MSGGPMGGRPGGRGAPAVQQNIPSTLLQDHENQRLFEMLGRKCLTLATAVVQLYLALPPG
AEHWTKEHCGAVCFVKDNPQKSYFIRLYGLQAGRLLWEQELYSQLVYSTPTPFFHTFAGD DCQAGLNFADEDEAQAFRALVQEKIQKRNQRQSGDRRQLPPPPTPANEERRGGLPPLPLH PGGDQGGPPVGPLSLGLATVDIQNPDITSSRYRGLPAPGPSPADKKRSGKKKISKADIGA PSGFKHVSHVGWDPQNGFDVNNLDPDLRSLFSRAGISEAQLTDAETSKLIYDFIEDQGGL EAVRQEMRRQEPLPPPPPPSRGGNQLPRPPIVGGNKGRSGPLPPVPLGIAPPPPTPRGPP PPGRGGPPPPPPPATGRSGPLPPPPPGAGGPPMPPPPPPPPPPPSSGNGPAPPPLPPALV PAGGLAPGGGRGALLDQIRQGIQLNKTPGAPESSALQPPPQSSEGLVGALMHVMQKRSRA IHSSDEGEDQAGDEDEDDEWDD Click to Show/Hide
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| 3D Structure | Click to Show 3D Structure of This Target | AlphaFold | ||||
| Drugs and Modes of Action | Top | |||||
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| Clinical Trial Drug(s) | [+] 3 Clinical Trial Drugs | + | ||||
| 1 | OTL-103 | Drug Info | Phase 3 | Wiskott-Aldrich syndrome | [2] | |
| 2 | Hematopoietic stem cell gene therapy | Drug Info | Phase 1/2 | Wiskott-Aldrich syndrome | [3] | |
| 3 | WASP gene therapy | Drug Info | Phase 1/2 | Wiskott-Aldrich syndrome | [1] | |
| Mode of Action | [+] 2 Modes of Action | + | ||||
| Replacement | [+] 1 Replacement drugs | + | ||||
| 1 | OTL-103 | Drug Info | [4] | |||
| Modulator | [+] 2 Modulator drugs | + | ||||
| 1 | Hematopoietic stem cell gene therapy | Drug Info | [5] | |||
| 2 | WASP gene therapy | Drug Info | [1], [6] | |||
| Target Regulators | Top | |||||
|---|---|---|---|---|---|---|
| Target-interacting Proteins | ||||||
| Target Affiliated Biological Pathways | Top | |||||
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| KEGG Pathway | [+] 9 KEGG Pathways | + | ||||
| 1 | Chemokine signaling pathway | |||||
| 2 | Adherens junction | |||||
| 3 | Fc gamma R-mediated phagocytosis | |||||
| 4 | Regulation of actin cytoskeleton | |||||
| 5 | Bacterial invasion of epithelial cells | |||||
| 6 | Pathogenic Escherichia coli infection | |||||
| 7 | Shigellosis | |||||
| 8 | Salmonella infection | |||||
| 9 | Choline metabolism in cancer | |||||
| Panther Pathway | [+] 1 Panther Pathways | + | ||||
| 1 | T cell activation | |||||
| PID Pathway | [+] 2 PID Pathways | + | ||||
| 1 | TCR signaling in naï | |||||
| 2 | ||||||
| Reactome | [+] 3 Reactome Pathways | + | ||||
| 1 | Generation of second messenger molecules | |||||
| 2 | Regulation of actin dynamics for phagocytic cup formation | |||||
| 3 | RHO GTPases Activate WASPs and WAVEs | |||||
| WikiPathways | [+] 7 WikiPathways | + | ||||
| 1 | TCR Signaling Pathway | |||||
| 2 | G13 Signaling Pathway | |||||
| 3 | Regulation of Actin Cytoskeleton | |||||
| 4 | Human Complement System | |||||
| 5 | Fcgamma receptor (FCGR) dependent phagocytosis | |||||
| 6 | Pathogenic Escherichia coli infection | |||||
| 7 | TCR signaling | |||||
| References | Top | |||||
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| REF 1 | ClinicalTrials.gov (NCT01347242) Gene Therapy for Wiskott-Aldrich Syndrome (WAS). U.S. National Institutes of Health. | |||||
| REF 2 | ClinicalTrials.gov (NCT03837483) A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome. U.S. National Institutes of Health. | |||||
| REF 3 | ClinicalTrials.gov (NCT02559830) Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy. | |||||
| REF 4 | Clinical pipeline report, company report or official report of Orchard Therapeutics. | |||||
| REF 5 | Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther. 2006 Oct;8(5):390-5. | |||||
| REF 6 | Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor. Haematologica. 2013 August; 98(8): 1300-1308. | |||||
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