Developing an initiative in stem cell editing for human genetic diseases.

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The recent development of genome editing using programmable nucleases has already transformed our ability to perform experiments in mammalian cells rapidly, at high throughput and with high precision. The next challenge is to apply this discovery to precisely edit and correct genetic defects in human stem cells in a new approach to the treatment of inherited diseases. In this proposal we aim to address the key preclinical hurdles in achieving this by editing haematopoietic stem cells from patients with severe haemoglobinopathies, with the goal of rapidly progressing this knowledge to early phase clinical trials.
Using flexible funding allocated to the University as a block grant, we will facilitate the development of this new initiative bringing together the considerable expertise and technical know-how in a wide range of interdisciplinary groups to address this focussed problem. We anticipate that genome editing will ultimately find application in a wide range of human diseases and the University of Oxford is fully committed to support its early development in blood diseases to establish the principles of this approach and the longer term application of this exciting new technology to a broad range of challenges in clinical medicine.