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Gene therapy hope for muscular dystrophy

3rd February 2006

New gene therapy techniques may be effective in treating patients with muscular dystrophy.

Originally developed to be used on people with skin diseases and haemophilia, the therapy could now dictate the future of muscular dystrophy treatments.

Researchers at the Stanford University School of Medicine believe that latest gene therapy can be used to place a gene into the body which will then go on to produce healthy dystrophin protein and help repair damaged muscle cells.

Although the research has so far only been conducted on mice, the authors of the study are confident that it will help to solve some of the previous problems with the therapy, such as introducing the genes into cells across the body and ensuring the production of protein is permanent.

Another issue is whether the dystrophin is spread along the length of the cell, warned Michelle Calos, associate professor of genetics, and does not leak out, preventing complete healing.

"If you have a single cell that's a foot long and you only correct a few inches, you've done very little," Ms Calos explained.

"Whereas if you correct it from end to end, you truly cure the disease in that cell.

"I think our approach has a lot of potential to overcome issues that have slowed the field of gene therapy," she concluded.