GENE EDITING WITH A MODIFIED ENDONUCLEASE

Smart overview of the Invention

An engineered nuclease with distinct binding and cleavage capabilities is being developed to enhance gene editing techniques. This modified endonuclease aims to alter cellular gene expression, providing a more precise tool for gene editing applications. The focus is on improving the efficiency and effectiveness of cellular therapies, particularly for treating diseases like cancer.

Background on Gene Editing Technologies

Gene editing technologies are pivotal in modern medicine, allowing for targeted modifications in genetic sequences. Current methods, such as CRISPR-Cas9, face limitations in specificity and efficiency. These challenges raise concerns about potential off-target effects that could lead to secondary health issues. The need for safer and more precise gene-editing tools is critical for therapeutic applications.

Chimeric Nuclease: TevSaCas9

The invention introduces a chimeric nuclease, referred to as TevSaCas9, which combines elements from I-TevI and Staphylococcus aureus Cas9. This nuclease is designed to target specific genes effectively, allowing for the deletion or modification of DNA sequences. By employing exogenous donor DNA, this tool enhances the precision of gene editing in immune and pluripotent cells.

Applications in Cellular Therapies

The modified endonuclease has significant implications for cellular therapies, particularly in immunotherapy and regenerative medicine. It can be utilized to engineer immune cells, such as T-cells, to better recognize and eliminate cancer cells. Additionally, the technology has potential applications in correcting genetic disorders by modifying stem cells for therapeutic purposes.

Methodology and Advantages

The methodology involves administering the chimeric nuclease to targeted cell populations without relying on viral vectors, thereby reducing safety concerns associated with traditional gene therapy approaches. The invention outlines specific sequences and modifications that enhance the nuclease's performance. By improving gene knockouts and introducing exogenous genes efficiently, this technology aims to revolutionize treatments for various diseases, offering a safer and more effective alternative to current therapies.