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A gene-editing method generates immunotherapeutic CAR T cells in the body

Azalea Therapeutics' dual-vector CRISPR system enables precise in vivo CAR T cell generation, achieving up to 40% CAR T cells in immune organs and clearing tumors in preclinical models.

Summary by Nature
Laboratory-engineered immune cells called CAR T cells provide effective treatment for some cancers. Progress is being made towards creating these cells in vivo. Laboratory-engineered immune cells called CAR T cells provide effective treatment for some cancers. Progress is being made towards creating these cells in vivo.

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Now, a team of scientists from the University of California at San Francisco (UCSF) has developed a method for reprogramming these cells directly into the body. The breakthrough, published this week in Nature magazine, is the first time that a long sequence of DNA has been integrated into a specific human T-cell site without ever having been removed from the body. “I think this is just the beginning of a great wave of new therapies that will be …

CAR-T cell therapy empowers the immune system to identify and attack cancer cells, but it requires immune cells to be created in a laboratory. However, a new method of genetic editing, tested in mice, allows the organism itself to generate them. The method was developed by a team headed by the University of California in San Francisco, USA, and has allowed the treatment of leukemia, multiple myeloma, and sarcoma in mice with the humanized immune…

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Nature broke the news in United Kingdom on Wednesday, March 18, 2026.
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