Gene therapy advance

Scientists believe that they have developed a way to increase the efficiency and safety of gene therapy. Usually, a  genetically altered virus is used to transport new genetic information into cells, by 'infecting' it with modified DNA. But viruses lose their effectiveness if they are repeatedly given to the same patient, as the immune system starts to attack them, and they can also cause unwanted side effects.

Now, scientists from Ohio's Case Western Reserve University and Copernicus Therapeutics, have found a way to compress DNA so that it is more easily absorbed into the nucleus of a cell, without the need for a virus. The DNA molecule is usually 'long and unwieldy', but compressing it into a small ball, 25 millionths of a millimetre wide, makes it easier to use and may allow modified genes to be given more easily to gene therapy patients. In laboratory studies, it appeared that the method was up to 6,000 times more effective than other methods.

Trials in 12 patients with cystic fibrosis have begun. People with cystic fibrosis have a fault in a single gene which means that thick mucus builds up in the lungs. The correct gene will be introduced in nasal cells, using the new method, to see if the procedure works. Later, it is hoped that it will be used to deliver genes directly to cells in the lungs. Robert Beall, president of the Cystic Fibrosis Foundation, said that there was excitement about the new procedure. He said 'everybody recognises that gene therapy could provide the cure for cystic fibrosis, and it is exciting that this is a non-viral approach'.