CAR-T therapy can produce spectacular remissions in blood cancers, but manufacturing it is slow and expensive: a patient's T cells must be extracted, genetically engineered in specialised facilities and reinfused. Researchers at Karolinska Institutet and the University of California, San Francisco, publishing in Nature with William Nyberg as first author, describe a method that performs the engineering inside the patient's own body.
The approach uses a two-component particle system: one element targets T cells using antibodies, while the other carries DNA that programs the cells to recognise tumours, together delivering CRISPR-Cas9 editing tools so the new receptor gene inserts at a precise site in the T-cell genome. In mice with humanised immune systems, a single injection cleared all detectable cancer in nearly all animals within two weeks, with the platform tested against aggressive leukaemia, multiple myeloma and a solid tumour model.
In vivo manufacturing, if it translates to humans, would address the cost and logistics that currently restrict CAR-T therapy to a fraction of eligible patients worldwide. The researchers are explicit that the technology must be scaled up for human use and that clinical trials are required to establish safety and efficacy, but the precision of gene-targeted insertion achieved inside a living organism represents a substantial technical landmark.