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 GTC0616
Trial ID JPRN-UMIN000011519
Disease Acute Myeloid Leukemia | Myelodysplastic Syndrome
Altered gene WT1
Therapeutic/Target gene Target gene
TherapyTCR-T cell
Treatment WT1 TCR-T cells
PhasePhase1
Recruitment statusNot Recruiting
TitleWT1-antigen specific TCR-gene transduced T lymphocytes transfer using MS3-WT1-siTCR vector for acute myelogeneous leukemia and myelodysplastic syndrome
Year2013
CountryJapan
Company sponsorEyetech Pharmaceuticals, Inc.
Vector information
Vectorretrovirus
ConstructpMS3-WT1-siTCR
Transgene/Inserted geneDNA encoding WT1235-243/HLA-A*24:02-specific TCR-α and TCR-β chains; interfering RNA constructs that specifically downregulate endogenous TCR
Vector production methodThe cells were stimulated with WT1 peptide. Brefeldin A was added, and they were then incubated with anti-CD8 monoclonal antibody (Becton Dickinson). The cells were stained intracellularly with anti-IFN-γ monoclonal antibody.

Clinical Result

Cohort1: dose level 1
Administration route None
Dosage 2E8 cells
Donor type autologous
Pts 3
Age Adult, Older_Adult
Outcome 1/3(PD)
Adverse reactions 2/3(Skin reaction); 1/3(Decrease of abnormal erythroblasts in PB); 1/3(Decrease of blasts in BM)
References DOI: 10.1182/blood-2017-06-791202
Cohort2: dose level 2
Administration route None
Dosage 1E9 cells
Donor type autologous
Pts 5
Age Adult, Older_Adult
Outcome 3/5(PD); 1/5(SD)
Adverse reactions 5/5(Skin reaction); 1/5(facial edema); 1/5(dermatitis); 1/5(arrhythmia); 1/5(stomatitis); 1/5(Decrease of blasts in BM)
References DOI: 10.1182/blood-2017-06-791202

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