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Researchers pioneer new gene therapy technique using natural repair process

x mutations in human immune cells makes us very optimistic that this therapy will work in several other diseases, such as sickle cell disease."

Sickle cell disease is a disorder in which blood cells carry a mutation in hemoglobin. The mutation causes the blood cells to change shape, preventing them from flowing through the blood vessels efficiently. One out of every 12 African-American individuals carries this mutation, and one out of every 500 African-American births is afflicted with disease.

In theory, blood stem cells with the sickle cell mutation could be removed, treated with the enzyme and the correct version of the mutant gene, and eventually given back to the patient, spawning the growth of healthy blood cells, Dr. Porteus said.


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Source:University of Texas Southwestern Medical Center at Dallas


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