Gene therapy useful in emphysema

BOSTON, Dec. 22 (UPI) -- A new gene therapy involving a single treatment could prevent the progression of inherited emphysema, scientists in Boston said.

Using mice, researchers at Boston University School of Medicine prevented the progression of Alpha-1 Anti-trypsin Deficiency, the most common inherited form of emphysema seen in young people, the university said in release. People who inherit the deficiency are predisposed to early emphysema and cirrhosis of the liver.

The researchers were able to deliver therapeutic genes to as much as 70 percent of a mouse lung's alveolar macrophages, a cell type that contributes to emphysema.

The lung macrophages carrying the therapeutic genes survived in the lung air sacks for the two-year lifetime of the mice and significantly improved the health of the mice, said Dr. Darrell Kotton, an associate professor of medicine and pathology who led the study.