Figure 1. A two-vector system is applied for in vivo CAR T-cell production, delivering CRISPR-Cas9 components that enable the engineered T-cells to target and destroy cancer cells.
CRISPR, a gene-editing tool, has improved cancer immunotherapy by amplifying an immune cell’s ability to recognize tumors, effectively preventing them from further developing. With the use of CRISPR-Cas9 technology, T-cells (white blood cells produced in the thymus) were engineered to produce Chimeric Antigen Receptor (CAR) -T cells, which target cancer cells. Usually, CAR-T therapy is performed in a laboratory setting, requiring immune cells to be removed from a patient, genetically modified, and then reinfused. Consequently, the therapy is time-consuming and expensive. However, researchers have found a way to genetically reprogram T-cells in vivo, allowing for direct immune cell modification within the patient to be done.
A two-vector system was developed to “deliver CRISPR-Cas9 ribonucleoproteins and a DNA donor template via enveloped delivery transporters and adeno-associated viruses” (Nyberg et al., 2026). By incorporating a CAR transgene into a particular locus receptive towards T cells, therapeutic levels of CAR T-cells were produced in vivo. This procedure was tested with humanized mouse models of B-cell aplasia, carrying blood and solid tumors. The engineered T-cells successfully attacked both the blood and solid tumors in the mouse models. Ultimately, the articles revealed the possibility of faster and more accessible CAR-T cancer treatments through CRISPR gene editing.
Links:
https://www.nature.com/articles/s41586-026-10235-x
https://pmc.ncbi.nlm.nih.gov/articles/PMC13096480/
This is extremely interesting and a huge step forward regarding CRISPR technology. The fact that this was accomplished in vivo is especially impressive and is sure to widen the scop of CAR-T related treatment of tumors. Good post!
ReplyDelete