Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D01QQC
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| Former ID |
DIB004820
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| Drug Name |
Hematopoietic stem cell gene therapy
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| Synonyms |
Hematopoietic stem cell gene therapy (Wiskott-Aldrich syndrome)
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| Indication | Wiskott-Aldrich syndrome [ICD-11: 3B62.0; ICD-10: D82.0] | Phase 1/2 | [1] | |
| Company |
Advantagene
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Wiskott-Aldrich syndrome protein (WAS) | Target Info | Modulator | [2] |
| KEGG Pathway | Chemokine signaling pathway | |||
| Adherens junction | ||||
| Fc gamma R-mediated phagocytosis | ||||
| Regulation of actin cytoskeleton | ||||
| Bacterial invasion of epithelial cells | ||||
| Pathogenic Escherichia coli infection | ||||
| Shigellosis | ||||
| Salmonella infection | ||||
| Choline metabolism in cancer | ||||
| Panther Pathway | T cell activation | |||
| Pathway Interaction Database | TCR signaling in naï | |||
| Reactome | Generation of second messenger molecules | |||
| Regulation of actin dynamics for phagocytic cup formation | ||||
| RHO GTPases Activate WASPs and WAVEs | ||||
| WikiPathways | TCR Signaling Pathway | |||
| G13 Signaling Pathway | ||||
| Regulation of Actin Cytoskeleton | ||||
| Human Complement System | ||||
| Fcgamma receptor (FCGR) dependent phagocytosis | ||||
| Pathogenic Escherichia coli infection | ||||
| TCR signaling | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT02559830) Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy. | |||
| REF 2 | Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther. 2006 Oct;8(5):390-5. | |||
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