Commercial Advantages
High efficiency gene editing in therapeutically-interesting cell types
Reduced requirements for donor DNA on a per-mole basis
Applications
Disease treatment
Gene therapy
Regenerative medicine
Drug testing and modeling
Gene-editing is an emerging therapeutic and regenerative medicine modality used to create new models for drug development and testing. Many gene editing workflows co-introduce reagents that create double strand breaks (DSB) in DNA and a homology donor containing a sequence that is copied into the genome region containing the break. The desired outcome of gene editing workflows is usually error-free incorporation of a homology donor sequence at the desired locus. A major limiting step in these workflows is utilization of the homology donor by the edited cells. Thus, a precise homology-directed repair (HDR) of the DSB has considerable therapeutic and biotechnical potential, but is still difficult to achieve.
Researchers at the University of California, Santa Barbara have dramatically increased HDR efficiency in human cell lines by modifying homology donors to include interstrand crosslinks. Modified donors are easy to manufacture in vitro and improve gene editing efficiency up to five-fold in cell lines and primary cells. Modified donors can be used to increase amounts of HDR, reduce homology donor quantities and consequent innate immune responses, and edit difficult cell types at useful levels. These enhancements to gene editing efficiency in therapeutically-interesting cell types can significantly impact the treatment of human disease and development of new models.
High efficiency gene editing in therapeutically-interesting cell types
Reduced requirements for donor DNA on a per-mole basis
Disease treatment
Gene therapy
Regenerative medicine
Drug testing and modeling