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Gene therapy may finally move out of the lab and into people's bloodstreams.
The European Medicines Agency has recommended the use of gene therapy for a rare genetic disease which leaves people unable to properly digest fats, BBC News reported. Before doctors can start treating the disease with gene therapy they must wait for it to be approved by The European Commission.
According to the BBC, lipoprotein lipase deficiency affects one in a million people. People suffering from lipoprotein lipase deficiency have damaged copies of a gene which is essential for breaking down fat. It leads to fat building up in the blood, abdominal pain and life-threatening pancreatitis (inflammation of the pancreas). So much fat can accumulate that the blood looks white rather than red.
Dr. Daniel Gaudet, a professor of medicine at the University of Montreal, told The New York Times, “It’s the equivalent of having a 10 percent cream in your bloodstream."
With gene therapy, patients are provided with correct copies of defective genes that cause genetic disorders, according to the New York Times.
UniQure, a Dutch company, developed a new gene therapy called Glybera. According to the LA Times, the treatment is injected into the patient's legs in a series of shots at one sitting. Gaudet tested the gene therapy on 27 people in clinical trials. The trails showed that the injections are long-lasting and provide at least partial control of lipoprotein lipase deficiency with no apparent adverse effects.
The European Commission has rejected Glybera three times before due to insufficient evidence of its effectiveness.
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