A new method of performing genetic therapy may lead to human trials as soon as next year. Groundbreaking research, carried out on mice by Clifford Steer and colleagues at the University of Minnesota Medical School in Minneapolis, has shown that it may be possible to fix faulty human genes, rather than to add new ones - the approach used in gene therapy trials to date. Instead of delivering new genes into the body by means of modified viruses, the new technique exploits the body's own mechanisms for repairing DNA.
A report in the latest Proceedings of the National Academy of Sciences describes how Steer's team used mice with a missing section of the gene for a liver enzyme that breaks down a toxic waste product called bilirubin. They injected the mice with molecules made of RNA and DNA designed to bind to the target gene, where enzymes responsible for DNA repair would fix the original gene fault.
A similar gene fault in humans leads to the rare disease, Crigler-Najjar syndrome, which has a high incidence in one Amish community in the American state of Pennsylvania. Steer's team believes that its new technique of gene repair may work in humans and could cure a syndrome such as Crigler-Najjar. Other experts hope that the technique will also be of use in combating other genetic conditions such as sickle cell disease and cystic fibrosis.
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