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Understanding research

Research can be complex and at times confusing.
A researcher wearing safety goggles and a lab coat conducts tests in a laboratory.

So we’ve brought together some useful resources to help you understand how research works, and to tell you about some of the important potential treatments that are emerging. Do get in touch to let us know what you think, and if you’d find any new resources helpful too.

What is exon skipping and how does it work?
In order to explain the concept of exon skipping, it is first necessary to explain how genes work and how mutations in the dystrophin gene can cause both Duchenne and Becker muscular dystrophy.
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AAV therapy in muscle-wasting conditions
Adeno-associated viruses (AAVs) are small viruses that infect humans and other primates but that are currently not known to cause any disease.
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Stem cells
Stem cells are considered to have great potential for treating numerous health conditions.
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The roles of animals in research
Animals play a vital role in understanding the causes of disease and in testing new treatments.
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What does a clinical trial coordinator do?
Clinical trial coordinators play an important role in the successful running of clinical trials. So much so that drug companies are unlikely to select a hospital to run a trial if a clinical trial coordinator is not in post.
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Genome editing
Genome editing is considered to have great potential for treating numerous health conditions, including muscle-wasting conditions.
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Glossary - A
Scientific jargon at a glance
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