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Limitations of conventional CAR-T therapy
Traditional CAR-T cell therapy involves extracting a patient’s immune cells, genetically modifying them in specialized labs, and reinfusing them. While highly effective, this process takes weeks and costs hundreds of thousands of dollars, limiting accessibility.
Direct in-body genetic modification
A study published in Nature describes a technique that allows precise genetic modifications to occur directly inside T cells. This enables the cells to produce chimeric antigen receptors (CARs) without leaving the body, avoiding random DNA integration seen in virus-based methods.
Dual-particle delivery system
The approach uses a dual-particle system with CRISPR-Cas9 gene-editing tools. One particle delivers the gene-editing machinery to T cells, while the second carries the DNA needed to produce CAR receptors. A molecular “on switch” ensures the DNA is activated only in T cells, and the particles are designed to evade immune system destruction.
Successful experiments in Mice
Experiments in mice with human-like immune systems showed treatment of multiple cancers, including acute lymphoblastic leukemia, multiple myeloma, and sarcoma tumors. Most animals showed complete elimination of detectable cancer cells within two weeks, alongside robust in-body generation of CAR-T cells.
Potential advantages over traditional methods
In-body CAR-T cells may expand more effectively and retain their functional properties better than lab-prepared cells. The method could reduce preparatory chemotherapy, lower costs, and accelerate patient access to treatment.
Next steps and clinical trials
Despite promising results, the technology is still in research stages. Further clinical trials are needed to confirm safety and efficacy in humans. The research team is working toward clinical use to make cancer treatments more accessible and cost-effective worldwide.