UniqueEDIT®
The UniqueEDIT® kit is designed to overcome challenges such as low cell membrane penetration and high off-target editing. By employing nanoscale needle-like structures, it directly delivers gene editing tools, including CRISPR-Cas9, into cells, enabling lossless delivery of Cas9 protein and sgRNA. The kit supports gene knockout, base editing, and epigenetic regulation, achieving efficient editing in hard-to-transfect cells such as immune cells (CAR-T, NK cells), stem cells (HSCs), and neurons. UniqueEDIT® provides a new solution that is highly efficient, precise, and reliable.
The Cas9 protein forms a complex with tracrRNA and crRNA, which guides the complex to DNA target sites containing a PAM sequence through crRNA complementarity. The HNH and RuvC domains of Cas9 then cleave the two DNA strands, enabling precise genome editing.
1、Gene editing in hard-to-transfect cells, such as optimizing CAR-T cells: Nano-needle delivery of CRISPR-Cas9 enables knockout of PD-1 or TCR genes, enhancing anti-tumor activity.
2、In vivo cancer therapy using CRISPR-Cas9: Potential application of CRISPR-Cas9 for directly treating cancers within the human body.
3、Curative approaches for genetic diseases, such as β-thalassemia: CRISPR editing of the HBB gene in patient-derived hematopoietic stem cells has allowed clinical trials (e.g., CRISPR Therapeutics’ CTX001) to achieve partial independence from blood transfusions.
Schematic of Gene Editing Mechanism
Illustration of In Vivo Cancer Therapy Using CRISPR/Cas9 by the University of Pennsylvania