Details of the Drug

General Information of Drug (ID: DMQWI7V)

Drug Name

Hematopoietic stem cell gene therapy

Synonyms

Hematopoietic stem cell gene therapy (Wiskott-Aldrich syndrome)

Indication

Disease Entry	ICD 11	Status	REF
Wiskott-Aldrich syndrome	3B62.0	Phase 1/2	[1]
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Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Wiskott-Aldrich syndrome protein (WAS)	TTE8T73	WASP_HUMAN	Modulator	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT02559830) Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy.
2	Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther. 2006 Oct;8(5):390-5.