In vivo site-specific engineering to reprogram T cells - PubMed
3 hours ago
- #T cell engineering
- #CRISPR-Cas9
- #cancer immunotherapy
- Engineered T cells with CAR or TCR have revolutionized cancer treatment and are being explored for other diseases.
- Current ex vivo manufacturing of engineered T cells is lengthy and costly, limiting accessibility.
- In vivo generation of CAR T cells could overcome these barriers but faces challenges with durability and specificity.
- A two-vector system using CRISPR-Cas9 ribonucleoproteins and a DNA donor template enables stable, cell-specific transgene expression.
- Optimized delivery methods allow for T cell-specific targeting and efficient gene integration.
- Therapeutic levels of CAR T cells were generated in humanized mouse models for B cell aplasia and various cancers.
- This approach offers a pathway to more efficient, precise, and widely accessible T cell therapies.