CRISPR-Based Delivery System Generates CAR T Cells In Vivo - Takeaways - MDSpire
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CRISPR-Based Delivery System Generates CAR T Cells In Vivo

A two-vector delivery system generates tumor-targeting CAR T cells directly inside the body, achieving complete responses in mouse cancer models

  • October 13, 2026

  • 3 min

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  • 1

    Researchers developed a two-vector system to deliver CRISPR-Cas9 machinery and DNA templates to generate CAR T cells directly in vivo.

  • 2

    The system uses CD3-targeted enveloped delivery vehicles and AAV-hT7, inserting the CAR transgene into the T-cell TRAC locus.

  • 3

    In humanized mice, the optimized system generated CAR-expressing cells comprising up to 19.7% of splenic T cells and completely depleted B cells.

  • 4

    The treatment produced complete responses in mouse models of B-cell acute lymphoblastic leukemia and multiple myeloma, and showed activity against sarcoma.

  • 5

    The platform remains preclinical, and immunodeficient mouse models limited assessment of immunogenicity, biodistribution, and safety.

Original Source(s)

CRISPR-Based Delivery System Generates CAR T Cells In Vivo

  • October 13, 2026

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