We have developed a caged truncated pre-tRNA-like crRNA catRNA enables precise and efficient gene ablation with an RNase-resistant property, compared to conventional crRNAs. The catRNA design has profound applications in gene editing of embryos and immune cells, such as T cells, wherein RNA electroporation is proven to be more efficient and less toxic than other gene delivery methods. Our technology demonstrates tremendous application potential in altering gene expression for the purpose of gene correction or disease treatment. Furthermore, it could generate a modified T cell much easier, particularly non-alloreactive T-cells for immunotherapy, thus enabling the establishment of standard and affordable adoptive immunotherapy strategies. Accumulating results have been collected including empowering this improved technology for treating disorders, such as cancer, infections or autoimmune diseases.
We have optimized a new version of CAS9 proteins CAS9-V2, whose efficiency improves several times as compared with wild type CAS9. This will largely ameliorate the perspectives of gene editing technology in diseases treatment by further decreasing its cost.
The CRISPR Cas9 technology are widely used for genome editing applications, however concerns such as double strand break and off-target side effects remain in debate. Recently, study conducted by Jussi Taipale from Karolinska Institute indicates that CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response, triggering elevated risk of cancer occurrence. In view of that, we harnessed base editor technology to specifically manipulate DNA or RNA at nucleotide level. This base editor technology exhibits great promise for precise treatment of cancers and genetic diseases.
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