Press

The GM Trust funds exon skipping project at Imperial College London

The Gavriel Meir Trust (GM Trust) and Imperial College London are pleased to announce that the Gavriel Meir Trust has funded the first year of a planned 3 year project entitled "Antisense oligonucleotides to restore the reading frame in Duchenne muscular dystrophy patients with out-of frame duplications".

Duchenne muscular dystrophy (DMD) is an X-linked fatal muscle wasting disorder that is associated with mutations in the gene that encodes a vital muscle protein called dystrophin. Without dystrophin the muscles are very fragile and undergo repeated cycles of damage that leads to loss of function. In about 10-15% of patients the mutations are large duplications of part of the dystrophin gene. Such duplications prevent the production of dystrophin by disrupting the reading frame. This project will test whether it is possible to modify these mutations using antisense oligonucleotides to restore the reading frame and so produce dystrophin and whether the larger than normal dystrophin will be functional. The project involves 3 laboratories, that of Professors Dominic Wells at Imperial College London, Francesco Muntoni at the Institute for Child Health and Steve Wilton of the Australian Neuromuscular Research Institute in Perth, Western Australia. Work on this project is expected to start in February 2008.