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Gene Therapy May Stall Inherited Emphysema

Posted Dec 22 2009 9:12am

A new type of gene therapy may help stop the progression of emphysema in young people who have an inherited form of the deadly disease.

Researchers say previous attempts to correct the gene mutation that predisposes young people to emphysema have failed to achieve lasting results.

But a new study shows a different approach that targets cells known as alveolar macrophages to deliver the gene therapy to the lungs of mice with this form of inherited emphysema was successful in treating the condition for two years.

Emphysema is a progressive lung disease that causes severe shortness of breath. There is no cure for the disease.

People born with a genetic mutation that causes a deficiency in alpha-1 antitrypsin are predisposed to an early form of emphysema as well as cirrhosis of the liver.

Researchers say this single gene defect makes the condition an ideal candidate for gene therapy, which would replace the defective gene with a normal one. But the problem until now has been finding the right cell in which to transfer the gene and deliver it to the lung.


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