A new gene therapy technique has been used to cure sickle cell anaemia in mice. Details of the study, undertaken by scientists from Harvard Medical School, US, are published in last week's issue of Science.
The scientists removed the bone marrow from mice with the sickle cell condition and transplanted new bone marrow infected with a genetically altered human immunodeficiency virus (HIV) into the mice. The virus carried a working copy of the haemoglobin gene, which replaced the defective gene that causes red blood cells to become sickle shaped. In the trials with mice, the researchers found that 99 per cent of the distorted cells were successfully corrected and the mice began to produce healthy, round red blood cells. They also noticed that other symptoms associated with the disease were lessened, including spleen enlargement, urinary problems and the dehydration of blood cells.
The success has raised hopes of a similar development in humans, but the technique will need to be refined before human trials can begin. The researchers must first show that the modified HIV virus is safe for humans.
Sources and References
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Gene therapy used to treat sickle cell in mice
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Scientists use gene therapy to correct sickle cell disease in mice
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Gene Gemisch Cures Sickle Cell in Mice
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