BBC NEWS : Gene therapy for blood disorder a 'success'

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BBC NEWS : Gene therapy for blood disorder a 'success'
« on: September 16, 2010, 10:07:48 AM »
Gene therapy has been used for the first time to treat an inherited blood disorder in what doctors say is a major step forward.

A man given pioneering treatment to correct a faulty gene has made "remarkable" progress, a US and French team has revealed.

Gene therapy is an experimental technique that manipulates genes in order to treat disease.

It has seen some successes, but also setbacks, including a patient's death.

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Beta thalassaemia is an inherited blood disorder that affects the body's ability to create red blood cells.

The first gene therapy trial was in an 18-year-old man with a severe form of the condition, who had been receiving regular blood transfusions since the age of three.

Stem cells from his bone marrow were treated with a gene to correct for the faulty one.

They were then transfused back into his body, where they gradually gave rise to healthy red blood cells.

Three years after the treatment, which took place in 2007, the man remains mildly anaemic, but no longer needs blood transfusions, doctors said.

The team, led by Philippe Leboulch, of Harvard Medical School in Boston, said: "At present, approximately three years post-transplantation, the biological and clinical evolution is remarkable and the patient's quality of life is good."


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