Basic Information

Gene symbol WT1 Synonyms AWT1, GUD, NPHS4, WAGR, WIT-2, WT-1, WT33 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description WT1 transcription factor

GTO ID GTC0151
Trial ID NCT01640301
Disease Acute Myeloid Leukemia
Altered gene WT1
Therapeutic/Target gene Target gene
TherapyTCR-T cell
Treatment WT1 TCR-T cells
HLAHLA-A*02:01
PhasePhase1|Phase2
Recruitment statusTerminated
TitlePhase I/II Study of Adoptive Immunotherapy After Allogeneic HCT With Virus Specific CD8+ T Cells That Have Been Transduced to Express a WT1-Specific T Cell Receptor for Patients With Relapsed AML
Year2011
CountryUnited States
Company sponsorFred Hutchinson Cancer Center
Other ID(s)2498.00|NCI-2011-03362|2498|2498.00|P01CA018029|RG9212029
Vector information
Vectorlentivirus
ConstructpRRLSIN.cPPT.MSCV/GFP.wPRE
Transgene/Inserted genesingle open reading frame consisting of the TCR β and TCRα chains
Regulatory elementa 2A element from the porcine teschovirus (P2A)
Viral genome modificationThe TCR donor C4 (TCRC4) gene construct was then inserted into the pRRLSIN.cPPT.MSCV/GFP.wPRE vector obtained from the lab of Richard Morgan after excising the green fluorescent protein (GFP) gene.
Vector production methodCells were stimulated with the HLA-A2-restricted EBV (EBV280-288 BMLF1 [GLCTLVAML]) peptide. On day +1 and day +2 of the stimulation, the responding cells were transduced using the pRRLSIN.cPPT.MSCV/TCRC4βP2ATCRC4α.wPRE lentiviral vector at a target multiplicity-of-infection of ≤3.

Clinical Result

Cohort 1
Administration route infusion
Donor type Allogeneic
Pts 15
Age Child, Adult, Older_Adult
Lymph depletion Yes
Outcome 8/15(CR); 3/15(PR); 1/15(PD)
Adverse reactions 8/15(All-cause mortality); 5/15(Blood and lymphatic system disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders)
References PMID: 31235963

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