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What defines a genetic disease and when might gene therapy be used?

March 18, 2020 @ 5:30 pm - 6:30 pm

Edinburgh Uni genetic disease and gene therapy
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What defines a genetic disease and when might gene therapy be used?

‘Let’s Talk About Health’ is all about advancing our knowledge of normal human and animal biology, and what goes wrong in disease.

Join us to hear about new research in our University that is increasing our understanding of diseases and providing new advances in treatment of both humans and animals. Guests will be able to talk to our young scientists about their research, and S4 and S5 pupils will have an opportunity to tour our labs before the talks at Edinburgh BioQuarter, or participate in a workshop before talks at Easter Bush. We look forward to seeing you there!

What defines a genetic disease and when might gene therapy be used?

David Fitzpatrick, Stuart Cobb, Queen’s Medical Research Institute

The term “genetic disease” can be used to describe a condition that is caused by a severe disruption of the sequence of an individual gene (known as a single gene disorder) or the influence of more subtle changes in many different genes on the chance you will develop a common disorder such as asthma or arthritis (known as complex genetic disorders). The former group are mostly very rare and the latter common. Although single gene disorders are rare they have been very important in helping us understand the understand the underlying mechanism of more common genetics disease. Single gene diseases are also leading the way in the application of novel genetic therapies.

Join us to hear about exciting new approaches for previously untreatable disorders that use engineered viruses to deliver gene therapy to the brain. This holds great promise for the treatment of severe genetic neurological disorders in both children and adults. To implement this exciting technology safely it is very important that we understand the consequence of the genetic changes in each rare disorder to determine whether we need to replace a gene product or remove it. This is an excellent paradigm for the future of personalised medicine.

This lecture is free and open to all.

Doors open 4.30pm with teas and coffees available.

Lab tours and/or workshops offered to S4-S6 school pupils (first-come-first-served) from 4.30pm -5.25pm.

Refreshments available after event.

For tickets head to Eventbrite 

>>How to get to Edinburgh BioQuarter


Details of the University of Edinburgh full privacy notice can be found here

We want to run a safe and accessible event and provide the best possible experience for you. By providing any information required on the booking form, you are giving the University consent to hold and process this data about you and to advise our suppliers of these details, under data protection regulations, for the purpose of running this event. By providing this information about your guest, you are confirming that you have their permission to share this data with the University for the same purposes. Any dietary and accessibility requirements are deleted following completion of the event.

Photography notice

This event may be photographed and/or recorded for promotional or recruitment materials for the University and University approved third parties. For further information please contact mvmpubliclectures@ed.ac.uk


Queen’s Medical Research Institute, The University of Edinburgh
47 Little France Crescent, Edinburgh BioQuarter
Edinburgh, Mid Lothian EH16 4TJ United Kingdom
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