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Breakthrough in the treatment of inherited genetic disease

Posted on 28 April 2016

Scientists at the Universities of York and Leiden have made a significant breakthrough in the treatment of an inherited genetic disorder which damages muscle and nerve cells in the body.

Gideon Davies

Pompe disease is caused by a defective gene that results in a deficiency of an enzyme called acid alpha-glucosidase (GAA) which causes progressive muscle weakness in people of all ages.

 The research team included Dr Liang Wu and Professor Gideon Davies of the Structural Biology Laboratory, Department of Chemistry, at York.

See http://www.york.ac.uk/news-and-events/news/2016/research/pompe-genetic-probes/ for the full article