
Researchers at the University of Edinburgh have perfected a genetic engineering strategy in mouse embryonic stem cells to replace large regions of mouse chromosomal sequences (>100kb) with the corresponding human syntenic region.
This state-of-the-art embryonic stem (ES) cell genetic engineering has the potential to achieve ‘humanisation’ of mice at the most advanced scale and level of sophistication yet attained to enable accurate functional analysis and disease modelling.
The RMGR technology combines aspects of various known technologies (gene targeting, Cre and FLP recombination) in a novel way to allow precise, reproducible and genetically selectable genomic replacement over a multi-kilobase size range that will permit the generation of improved mouse models for studying human disease.
This method in collaboration with the Medical Research Council’s Molecular Haematology Unit in Oxford has been exemplified by generating a mouse that produces only the human α globin chains. It has also generated an accurate model of the human disease α thalassemia resulting from a major regulatory mutation.
The technology has a priority UK patent application filed and is available for licensing.
The University of Edinburgh is seeking commercial partners for both collaboration and licensing opportunities of this technology.