US20260152766
2026-06-04
Chemistry; metallurgy
C12N15/907
The patent details a novel approach for delivering and utilizing the CRISPR-Cas system to target and modify specific genetic sequences associated with various disorders and diseases. Central to this invention is the use of nanoparticle delivery formulations, which facilitate the precise targeting of CRISPR components to desired genomic loci. This method enhances the specificity and reduces the toxicity of CRISPR applications, enabling effective editing or modification of target sites to improve disease conditions.
The invention emphasizes the use of nanoparticles to deliver CRISPR-Cas components, ensuring efficient transport to specific cells or tissues. These delivery systems can be engineered to carry vectors encoding CRISPR components, such as guide RNAs and Cas enzymes. This targeted delivery is crucial for directing CRISPR complex formation within eukaryotic cells, thereby enhancing the precision of genetic modifications while minimizing off-target effects.
The CRISPR-Cas system described in the patent has broad applications in gene editing, gene therapy, drug discovery, and disease diagnosis. By modifying target polynucleotides in various cell types, including endothelial, skin, heart, muscle, and lung cells, the system can be used to delete, insert, or alter genes. This versatility makes it a valuable tool for advancing medical and biotechnological research, as well as for developing new therapeutic strategies.
The invention outlines methods for using CRISPR-Cas components via nanoparticle delivery to modify genetic sequences in organisms. These methods include delivering engineered compositions containing CRISPR-Cas system chimeric RNA polynucleotide sequences and polynucleotide sequences encoding CRISPR enzymes. The system ensures that the guide sequence directs the CRISPR complex to the specific target sequence, enabling precise genome editing.
The CRISPR enzyme, particularly the Cas9 enzyme, is a key component of the system. The patent specifies the use of type II CRISPR enzymes, such as those derived from S. pyogenes or S. aureus, due to their efficiency and specificity. These enzymes can be delivered constitutively or via nanoparticles, enhancing the system's adaptability for various applications. The invention also includes potential modifications to these enzymes to improve their functionality and broaden their application scope.