£2.5 million HICF award to Dr Matthew Wood for research into Duchenne muscular dystrophy


A £2.5 million HICF award to Dr Matthew Wood of the Department of Physiology, Anatomy and Genetics and Somerville College will fund research into Duchenne muscular dystrophy (DMD), the most deadly form of the disease. It is caused by damage to the dystrophin gene and affects one in every 3,500 newborn boys.

Dr Wood and colleagues at the MDEX Consortium – a UK-based translational medicine network of neuromuscular disease experts working to turn scientific findings into drugs and other forms of healthcare – have successfully trialled a treatment using antisense oligonucleotides (AOs) – DNA-based drugs that can repair the gene and restore functional dystrophin to the muscles of DMD patients.

Currently the AO drug is only moderately effective in skeletal muscle and does not work on heart muscle.

Led by Dr Wood, the group is now developing a new generation of AO drugs which will dramatically improve the delivery and restoration of dystrophin by all muscle. The treatment will be tested in a clinical trial on nine DMD patients, starting in 2013.




Does anyone knows more about this work.

Whether Dr Wood is working with any pharma company?

How is this work different from current AVI formulation.

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It doesnot mention who will benefit from it? Will all the DMD boys will be benefit from this? Thanks.

I think they are working on improved delivery PPMO type compounds (better delivery to heart etc.). Not sure what exons they plan to skip in this first small trial.

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