103608Advanced Tools for Human Genomic TherapeuticsClosedCollaborative R&DISCFOur DNA defines how we live, how we age, how we die, and most of the illnesses we suffer along the way. The last 20 years have seen us decode human DNA (our "genome"), boosting our understanding of disease and enabling "personalised medicine" tailored to the individual. But understanding the genome is simply not enough. Treatments still target the complex downstream consequences of DNA mutations, not the root cause of the illness itself: we've got better at chasing horses, but still can't bolt the stable door. The next decades will see a re-focussing of therapeutic effort: we must start to tackle disease at the level of DNA. Inherited disorders like cystic fibrosis will be tackled not with partially-effective palliative therapies, but by precise DNA editing to correct the causative error. Cancers will be cured not by the blunderbuss approaches of chemo- or radiotherapy, but by genomic targetting of cells with cancerous DNA mutations. Diseases such as Alzheimer's will be prevented by learning from those lucky few whose genes already protect them - and using genome editing to offer that same protection to the majority. Scientists and ethicists are rightly cautious about genomic intervention: current tools are imprecise and carry grave risks that will continue to become more apparent. Mote Research has identified a new approach to genome editing that is exquisitely precise and applicable therapeutically as well as for research. We seek funding to build on this early work, to secure critical IP, and to complete early trials of this technology.