Overview
- Nyberg et al. report in Nature that a UCSF‑led team reprogrammed T cells inside mice to express cancer‑targeting CARs using site‑specific gene editing.
- A dual‑particle system delivered CRISPR‑Cas9 to circulating T cells via anti‑CD3 targeting and inserted CAR DNA at a defined genomic activation site.
- In aggressive leukemia models, a single injection generated CAR‑T cells comprising up to 40% of immune cells and cleared all detectable disease in nearly all mice within two weeks.
- The approach also showed activity against multiple myeloma and a solid sarcoma, and the in vivo–engineered cells appeared more stem‑like than lab‑manufactured counterparts, according to the authors.
- The work remains preclinical and will require human testing; the team has filed patents and founded Azalea Therapeutics, and a related News & Views article discloses an author’s ties to NanoVation Therapeutics.