Sickle-Cell Cure in Mice

Reported in Gene Trial

Scientists have cured sickle-cell disease in a strain of mice by using a powerful gene therapy method that can replace nearly all diseased blood cells with healthy ones. Patients with sickle-cell disease have blood cells that deform when oxygen levels are low, creating painful blockages of blood vessels and producing organ damage.

Researchers from Harvard University and MIT reported in the Dec. 14 issue of Science that they used a modified virus to treat the disease. The virus carried a normal copy of a gene that allows blood cells to carry oxygen without deforming. When the researchers injected the virus into the mice, the new gene quickly replaced the defective one in bone marrow cells, where new blood cells are made. Much more work is required before the technique can be tested in humans.

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Compiled by Times medical writer Thomas H. Maugh III