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Genome editing: a scientific revolution

A distinguished panel of experts from four leading bioscience research institutes come together during this year’s Cambridge Science Festival to discuss and respond to public questions about the current and future applications of genome editing – a process by which sections of DNA are removed or replaced.

Human genomes contain natural errors or mutations. Most are relatively harmless and underpin evolution but some cause life-threatening diseases. In a few cases, patients have been treated by conventional gene therapy, where new genes are incorporated using a carrier virus to counteract defective genes. However, so far few patients have been treated by genomic editing.

This could all change now that new and much publicised genome editing tools and techniques, such as CRISPR, are being applied in research areas as diverse as horticultural crop breeding and infectious and genetic diseases. Crucially, these editing tools have arrived in the midst of a second revolution – a revolution in our ability to sequence large genomes faster, using techniques developed in Cambridge.  

During the , members of the audience have the opportunity to question a range of opinions and expertise from active researchers who will introduce the latest scientific developments in this field. The event is co-hosted by four leading bioscience research institutes:; ; ; and The ����������̳. The panel includes:

, Group Leader in Epigenetics at the ����������̳ and Affiliate Group Leader at the Stem Cell Institute. The Rugg-Gunn group investigate how epigenetic processes are involved in regulating stem cells and the early stages of mammalian development. This knowledge provides insight into how the different cell types of the body are formed and maintained. The group’s recent research uses genome editing to delete epigenetic modifiers (proteins that switch genes on and off) in human embryonic stem cells in order to understand what those genes do in human development and how they might control cell differentiation and identity. 

In discussing the group’s recent work, Dr Rugg-Gunn said: “Gene editing technologies enable the removal of selected genes with high efficiency and accuracy. By deleting epigenetic modifiers in human embryonic stem cells, our research has uncovered how these key proteins work in order to carefully control gene activity during the earliest stages of human development. These findings are also important for refining approaches to control embryonic stem cell specialisation towards desired cell types that could be used for regenerative medicine, for instance after a heart attack.
“The emergence of these new exciting technologies raises the possibility of a wide range of applications that could lead to a better understanding of human biology and disease.”

Professor Azim Surani, Director of Germline and Epigenomics Research at the Gurdon Institute and Affiliate group leader at the Stem Cell Institute. Professor Surani is a high-profile researcher on the creation of precursor human sperm and eggs from stem cells and has published widely on this area. Gene editing of the human germline – those cells that form the sperm and eggs and, from a fertilised egg, will generate every cell in the human body – is different from other types of genetic editing because changes in those cells will be inherited by future generations, to become a permanent change in the human make-up.

Professor Ludovic Vallier, Group Leader at the Stem Cell Institute and the Sanger Institute. His research focuses on how stem cells become the pancreas, lung, gut and liver cells. Some of the team’s work uses genome editing technology to correct or inhibit the genes responsible for diseases such as cystic fibrosis and liver disease. This is currently being proven in ‘in vitro models’ – meaning cells outside of a living organism. Professor Vallier will discuss new areas where genome editing techniques are helping research to progress rapidly and how he thinks this may lead to real treatments for cystic fibrosis, diabetes and liver diseases.

Speaking about his team’s current research, Professor Vallier said: “Our team is able to make stem cells (the body’s master cells that can turn into almost any type of cell in the body) from blood cells of cystic fibrosis patients, which in itself is quite remarkable. We are now able to use gene editing technology to correct the disease causing mutations in these stem cells and allow them to develop into the cells that line the lung and the liver. This is allowing us to study CF in a dish and to develop platforms for drug screening. The possibility to produce functional lung and liver cells from CF patients represents a first step toward new regenerative medicine treatments.”

Dr Marcus Lee, Group Leader at the Sanger Institute. The Lee research group use genome editing (amongst other techniques) to investigate parasite drug resistance – particularly in the human malaria parasite Plasmodium falciparum. They are discovering key information about how parasites mutate and develop resistance.

Other related events during the Science Festival include:

• A second co-hosted by the same four bioscience research institutes  tackles personalised medicine including how stem cells contribute to diagnostics and drug development
• Dr John Perry the role of genetics in health and disease, with a particular focus on human growth and reproduction.
• Scientists from Illumina how they combine engineering, chemistry, genetics, and informatics to revolutionise the way we look at our DNA, and how they are working with the NHS to change the way disease is diagnosed.
• Professor Ashok Venkitaraman from the Medical Research Council’s Cancer Unit recent scientific advances that promise to transform our ability to translate biological knowledge into new medicines.

Since its launch in 1994, the has inspired thousands of young researchers and visitor numbers continues to rise; last year, the Festival attracted well over 45,000 visitors. The Festival, one of the largest and most respected, brings science, technology, engineering, maths and medicine to an audience of all ages through demonstrations, talks, performances and debates. It draws together a diverse range of independent organisations in addition to many University departments, centres and museums.

This year’s Festival sponsors and partners are Cambridge University Press, AstraZeneca, MedImmune, Illumina, TTP Group, Science AAAS, BlueBridge Education, Siemens, ARM, Microsoft Research, Redgate, Linguamatics, FameLab, ����������̳, Wellcome Genome Campus, Napp, The Institute of Engineering and Technology, St Mary’s School, Anglia Ruskin University, Cambridge Junction, Addenbrooke’s Hospital, Addenbrooke’s Charitable Trust, James Dyson Foundation, Naked Scientists, Hills Road Sixth Form College, UTC Cambridge, British Science Week, Alzheimer’s Research UK, Royal Society of Chemistry, Cambridge Science Centre, Cambridge Live, and BBC Cambridgeshire.

Image copyright: Genome Research Ltd.