Genetic diseases arise from a wide spectrum of mutations, such as point mutations, insertions, deletions, and chromosomal rearrangements—each presenting unique challenges for therapeutic intervention. To address these diverse needs, a versatile set of genome editing tools must be developed and optimized for specific disease contexts.

 

However, genome editors alone are not sufficient. The outcomes of genome editing are heavily influenced by the target cell type and the DNA repair pathways that the cell naturally employs. Therefore, understanding how host cells respond to editing events is critical for improving the efficiency, accuracy, and safety of genome editing strategies.

 

Moreover, successful in vivo genome editing requires precise delivery of the editing machinery to the relevant tissues and cell types. This necessitates the development of targeted delivery systems—both viral and non-viral—tailored to the biology of the affected organ.

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To address these challenges, the Liu Lab focuses on three primary research areas:

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Discovery of Novel Genome Editing Tools  

Expanding the molecular toolkit to precisely correct a broad range of genetic mutations.

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Understanding Cellular Responses and DNA Repair Mechanisms in The Context of Genome Editing  

Elucidating how different cell types respond to genome editing and how DNA repair pathway choices influence outcomes.

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Targeted Delivery of Genome Editors for Disease Treatment  

Employing viral and non-viral delivery platforms for organ- and cell-specific genome editing for therapeutic applications.