Hope for people with Duchenne Muscular Dystrophy
A new treatment has been used to treat the mouse equivalent of Duchenne Muscular Dystrophy (DMD). Attempts to treat DMD with gene therapy have so far failed because the faulty gene is exceptionally large and difficult to get into the appropriate muscle fibres. Researchers at the MRC’s Clinical Sciences Centre developed a new technique, called antisense therapy, that uses 'molecular camouflage' to blind the cell's protein manufacturing machinery to DMD mutations. This allows the key parts of the defensive protein to be made so it works almost normally, relieving many symptoms of the disease.
Nature Medicine 2003; 9: 1009-1014