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

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Queen's Medical Research Institute, The University of Edinburgh

47 Little France Crescent

Edinburgh BioQuarter

Edinburgh

EH16 4TJ

United Kingdom

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

About this Event

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.

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Date and Time

Location

Queen's Medical Research Institute, The University of Edinburgh

47 Little France Crescent

Edinburgh BioQuarter

Edinburgh

EH16 4TJ

United Kingdom

View Map

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