Tissue-Specific Genome Engineering Using CRISPR-Cas9

Patent Status

Additional Patents Pending

Brief Description

Delivering gene-editing agents safely and effectively has long been a challenge in modern medicine. Traditional methods using viral vectors introduce risks such as insertional mutagenesis, hepatotoxicity, and transient therapeutic effects due to immune responses. UC Berkeley researchers have developed a groundbreaking tissue-specific genome engineering system utilizing CRISPR-Cas9, offering a safer and more precise alternative for gene therapy.

UC Berkeley researchers and others have created compounds, compositions, uses thereof the combines the cutting-edge CRISPR technology with advanced targeting mechanishms for the treatment of diseases, conditions and/or disorders, and uses thereof as asialoglycoprotein receptor (ASGPR) targeting agents.

Suggested uses

• Gene editing 
• Gene delivery 

Advantages

• By leveraging ASGPR targeting agents, this approach ensures precise uptake by hepatocytes and other specific tissues.
• Avoids the risks associated with viral vectors, reducing toxicity and immune complications.
• By delivering gene-editing agents in protein form rather than DNA or RNA, the technology maximizes therapeutic potential while minimizing unwanted genetic disruptions.
• Overcomes the limitations of receptor-mediated endocytosis to ensure the therapeutic agents effectively reach their intended subcellular locations.