Cystic fibrosis mice cured in the womb

摘要

Mice have been cured of cystic fibrosis after receiving a novel form of gene therapy in the womb, raising hopes that the disease might be prevented in humans. The success of the gene therapy also challenges orthodox theories about the cause of the disease. Janet Larson of the Ochsner Medical Foundation in New Orleans, who treated the mice, is confident that the same approach will work in humans. The hereditary disease causes premature death by clogging the lungs and gut with mucus. About 6500 people in Britain have cystic fibrosis. "We are very optimistic that we can prevent this disease," says Larson. The work is reported in The Lancet (vol 349, p 619).