Gene therapy corrects sickle cell disease in mice, Science authors report

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A new gene therapy method corrects sickle cell disease in mice, and may suggest future therapies to treat the genetic disease in humans. The method, developed by an international research team, is described in the 14 December issue of the journal, Science.

The therapy counteracts the faulty gene that causes red blood cells to "sickle" or deform, by transferring an anti-sickling variant of the gene to bone marrow with a viral delivery system. Once there, the anti-sickling gene incorporates itself into the stem cells that give rise to red blood cells.

In two mouse models, the new gene was rapidly expressed in 99 percent of all circulating red blood cells, preventing sickling and other signs of the disease, says study author Philippe Leboulch of the Harvard Medical School and Massachusetts Institute of Technology.

Leboulch says that there are still several obstacles to overcome before the therapy can be tested in humans, but that the method is "clearly corroborating evidence that these types of vectors based on lentiviruses are highly efficient with stable expression."

Sickle cell disorders are most common in individuals of African, Mediterranean, Indian, and Middle Eastern descent, and one in every 13 African Americans carries the sickle cell trait, according to Leboulch. The disease is caused by a single nucleotide mutation in the human beta globin gene.

Why does a mutation of the beta globin gene cause disease? Red blood cells need oxygen. In healthy people, oxygen is carried to red blood cells by hemoglobin, a molecular complex. Beta globin plays a role in getting oxygen to red blood cells, too, because it contributes two protein "chains" to hemoglobin. When a person inherits the sickle cell mutation from both parents, he or she manufactures an abnormal version of hemaglobi

Contact: Lisa Onaga
American Association for the Advancement of Science

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