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Fli-1 knockout CAR T-cell

Development stage
Preclinical
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

Fli-1 knockout CAR T-cell is an engineered cell therapy designed to enhance anti-tumor immunity. It involves genetically modifying CD19-directed human CAR T cells using CRISPR technology to knock out the Fli-1 gene, which is a transcription factor. The absence of Fli-1 in these CAR T cells leads to elevated activation, proliferation, and cytolytic function, along with reduced expression of exhaustion markers such as PD-1, TIM-3, and LAG-3. This modification aims to improve the efficacy and persistence of CAR T-cell therapy against various cancers. Preclinical studies have demonstrated enhanced tumor control and extended survival in models of melanoma and B cell lymphoma.

02

Targets

CD19 (B lymphocyte antigen CD19)FLI1 (Flightless I actin-binding protein)

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