Today we know a lot about the genetic basis of disease traits. For example more than 5,000 diseases, affecting more than 250 million people globally, are caused by changes in just one gene. These include conditions like cystic fibrosis, Huntington’s disease and sickle cell anaemia.
But until recently there has been a gap between diagnosis of a genetic disease and the promise of a cure. Now gene-editing tools like CRISPR are offering new hope. This talk will illustrate how CRISPR can be used to fix disease-causing broken genes in adult cells. Controversially, in 2018, it was used to create gene-edited twin baby girls in the CRISPR baby scandal, opening a Pandora’s Box of the potential to create inheritable genetic changes.
The complex technical, ethical and social issues involved in gene-editing in embryos, will be explored in this captivating talk.
Dr Hilary Sheppard is a Senior Lecturer in Stem Cell and Developmental Biology. Her interest in understanding how cells become different to one another has led to clinical applications of gene-editing. In one project, her team are working to enhance cancer therapies using a patient’s own immune cells. In another, they are working to develop gene-edited patient-specific skin sheets for people with a fragile skin condition called epidermolysis bullosa, also known as butterfly skin.