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Gene therapy roots out emphysema in clinical tests

Category: General Health

Genetic testing for mutations which are responsible for causing emphysema may lead to a single-treatment therapy which provides lifetime protection against the progression of the lung disease, according to recent research.

In the background information to a study published in the December 21 online issue of the Journal of Clinical Investigation, scientists from the Boston University School of Medicine identified a mutation in the alpha-1 anti-trypsin gene as the most common cause of emphysema in young adults, HealthDay News reports.

During their study, the team of researchers was able to develop a system that selectively delivered therapeutic genes to as many as 70 percent of the lung's alveolar macrophages, the white blood cells which play a role in emphysema development.

"The lung macrophages carrying the therapeutic gene survived in the lungs' air sacs for the two-year lifetime of the treated mice following a single injection of the [system] we had engineered," said the study's senior author Dr Darrell Kotton, co-director of the Center for Regenerative Medicine at Boston University.

According to Dr Andrew Wilson, the study clarifies that lung macrophages may be targeted for further studies on gene therapy.
The Centers for Disease Control and Prevention reports that emphysema kills more than 12,000 Americans each year.

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