DMD research

World Duchenne Day: Two New Gene Editing Projects

Today is World Duchenne Awareness Day - a day when sixty nine patient organisations and thousands of boys and young men living with Duchenne from all over the world, come together with one voice, to raise awareness of this cruel disease.  

Please visit the WDAD website to read the thought-provoking photoblogs of boys and young men, their carers and families. 

To mark this important day, we are announcing two exciting gene editing projects – one in London and one in Toronto - as part of our commitment to fund global research.

Firstly, we are investing CAN$250,000 to fund the work of Dr Ronald Cohn, Chief of Clinical and Metabolic Genetics at Toronto's Hospital for Sick Children (pictured below). His research involves taking CRISPR, a genome-editingtechnology that allows for the precise manipulation of DNA in the nucleus of any cell, one step further towards clinical trial.

Secondly, together with Muscular Dystrophy UK, we are funding a £180,000 three-year project by Professor Francesco Muntoni, of University College London. His work aims to remove an extra copy of the dystrophin gene, which causes Duchenne in around 15% of cases.

Both projects are part of the Duchenne Children’s Trust world-wide research portfolio that aims to make a difference to this generation of boys and young men with Duchenne Muscular Dystrophy

Published on 7 September 2015

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