Reprogrammed stem cells cure sickle cell anaemia

Published on December 9, 2007 at 6:20 PM · No Comments

Scientists in the U.S. have made a breakthrough in the fight against sickle cell anaemia, a blood disease caused by a defect in a single gene.

Sickle cell anaemia is a serious condition in which the red blood cells can become sickle-shaped instead of smooth and round; they do not move easily through the blood because they are stiff and sticky and can form clumps in the blood vessels.

These clumps block the blood flow in the blood vessels and can cause pain, serious infections and damage to limbs and organs.

Sickle cell anaemia affects millions of people worldwide but is most common in people whose families come from Africa, South or Central America (especially Panama), the Caribbean, Mediterranean countries (such as Turkey, Greece, and Italy), India and Saudi Arabia.

Sickle cell anaemia affects about 70,000 people in the United States mainly African Americans and around 2 million Americans have the sickle cell trait.

The scientists created the stem cells from cells extracted from the skin of mice; by injecting 4 genes into these new stem cells they were able to reprogramme them.

When the reprogrammed cells were reinjected into mice with sickle cell anaemia, the mice were cured.

In order to turn the skin cells into master cells the four genes were delivered using a type of virus called a retrovirus.

The team at the Whitehead Institute of Biomedical Research in Cambridge, Massachusetts, say they have shown that the cells 'pluripotent stem cells' (iPS), have the same potential for therapy as embryonic stem cells, but without the ethical and practical issues.

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