What type of study is this?

This is a Human Clinical Trial study (also classified as: In Vitro Study, Pre-Registered Trial).

What question did this study set out to answer?

Investigate the effectiveness of triple base-edited CAR T cells targeting GM-CSF, IL3, and FLT3LG in acute myeloid leukemia.

December 8, 2025Blood

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Demonstrates increased efficacy of CAR T cells with triple gene editing in acute myeloid leukemia, indicating cytokines can be targeted.

Investigate the effectiveness of triple base-edited CAR T cells targeting GM-CSF, IL3, and FLT3LG in acute myeloid leukemia.
Donor T cells were engineered with a chimeric antigen receptor (CAR) targeting CD70 and CD33.
CAR T cells were edited to be deficient in GM-CSF, IL3, and FLT3LG using CRISPR cytosine base editing.
Editing efficiency was assessed via flow cytometry, western blot, ELISA, and DNA sequencing.
Triple-edited CAR T cells showed improved anti-leukemia potency and in vitro proliferation compared to controls.
Editing efficiencies achieved were 85% for GM-CSF, 55% for IL3, and 75% for FLT3LG.
scRNAseq revealed high levels of GM-CSF and IL3 in CAR T cells post-treatment, indicating their role in leukemia progression.

Bratt et al. (2025) studied this question.

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