US20260022392
2026-01-22
Chemistry; metallurgy
C12N15/746
The patent application describes advanced systems, methods, and compositions for manipulating genetic sequences using the CRISPR-Cas technology. It focuses on the development of vector systems that encode components of the CRISPR complex, aimed at facilitating precise sequence targeting and modification in eukaryotic cells. These systems are designed to introduce specific mutations and direct the formation of CRISPR complexes, enhancing the accuracy and efficiency of gene editing processes.
The invention includes a vector system comprising multiple vectors, each playing a role in the CRISPR complex assembly. Key components include regulatory elements linked to guide sequences and CRISPR enzymes. These elements work together to ensure sequence-specific binding and activity within the target cells. The CRISPR complex is guided by RNA molecules to target specific DNA sequences, making the technology versatile for various genetic engineering applications.
The CRISPR-Cas system offers a significant improvement over previous genome-editing technologies by eliminating the need for customized proteins to target specific sequences. Its ability to be programmed with short RNA molecules simplifies the process, making it more accessible and scalable. This system is particularly beneficial for applications in synthetic biology, biotechnology, and medicine, where precise genetic modifications are crucial.
The patent emphasizes the importance of engineering and optimizing the CRISPR-Cas system to enhance its functionality and minimize potential side effects. By incorporating nuclear localization sequences and optimizing codon usage, the invention ensures effective targeting and activity of the CRISPR complex within eukaryotic cells. This optimization is critical for achieving high specificity and efficiency in genome editing tasks.
The vector systems described can include various types, such as plasmids and viral vectors, each capable of transporting and expressing the necessary genetic components. These vectors are designed to replicate autonomously or integrate into the host genome, depending on the application. The flexibility in vector design allows for tailored solutions to meet specific genetic engineering needs, enhancing the overall utility of the CRISPR-Cas technology.