CLL1-CAR-T CELL, AND PREPARATION METHOD THEREOF, AND USE THEREOF

Smart overview of the Invention

The CLL1-CAR-T cell invention introduces a chimeric antigen receptor (CAR) designed to target CLL1, a molecule prominently expressed in myeloid cells and acute myeloid leukemia (AML) cells. The CAR includes a single-domain antibody, a hinge region, a transmembrane region, and an intracellular signaling region. The single-domain antibody is derived from a specific amino acid sequence, enabling the CAR-T cells to secrete the effector molecule IFN-γ, efficiently kill CLL1+ target cells, and demonstrate strong anti-tumor activity in vivo.

Technical Details

The CAR-T cells are engineered to express a chimeric antigen receptor comprising several key components: a single-domain antibody, a hinge region, a transmembrane region, and an intracellular signaling region. The intracellular signaling region includes the co-stimulatory domain 4-1BB and the activation domain CD3ζ, which are crucial for initiating immune responses. The hinge and transmembrane regions are derived from CD8a sequences, contributing to the stability and functionality of the CAR.

Biological Materials

The invention also encompasses various biological materials related to the CLL1-CAR-T cells. These include nucleic acid molecules encoding the chimeric antigen receptor, expression cassettes, recombinant vectors, and recombinant microorganisms. These materials facilitate the expression and function of the CAR, enabling the targeted immunotherapy for AML and potentially other related diseases.

Potential Applications

The CLL1-CAR-T cells present a promising therapeutic approach for treating AML by targeting the CLL1 antigen. Due to CLL1's unique expression pattern on myeloid cells and AML cells, these CAR-T cells offer a targeted method to combat the disease, potentially overcoming the limitations of traditional therapies like chemotherapy and stem cell transplantation. The invention's focus on CLL1 provides a novel avenue for immunotherapy, with broad prospects for clinical application.

Future Implications

The development of CLL1-CAR-T cells represents a significant advancement in the field of cellular immunotherapy. By addressing the challenges associated with targeting myeloid cells, this invention could lead to more effective treatments for AML and improve patient outcomes. Continued research and development could expand the applications of this technology to other hematological malignancies and enhance the efficacy and safety of CAR-T cell therapies.