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Boston University study of Alpha-1 mice shows promise that gene therapy may improve COPD in Alphas
A new type of gene therapy may help stop the progression of emphysema (COPD) in people who have Alpha-1 Antitrypsin Deficiency, sometimes called “genetic COPD”.
Boston University researchers say previous attempts to correct the Alpha-1 gene mutation that predisposes people, including non-smokers, to COPD, have failed to achieve lasting results.
But a new study shows a different approach that targets cells known as alveolar macrophages (white blood cells) to deliver the gene therapy to the lungs of mice was successful in treating the condition for two years—the full lifespan of the mice.
“We applied this novel approach to achieve sustained in vivo expression of normal human alpha-1 antitrypsin (hAAT) protein at levels able to ameliorate emphysema in mice,” said senior author Darrell Kotton, MD, an associate professor of medicine and pathology and co-director, Center for Regenerative Medicine at BUSM.
“The progression of emphysema in mice exposed to elastase was significantly improved by the gene therapy as evidenced by improvements in lung compliance and alveolar size,” said Andrew Wilson, MD, lead author of the study and an assistant professor of medicine at BUSM.
“Our results challenge the dogma that lung macrophages are short-lived and suggest these differentiated cells as a target cell that may be considered for in vivo gene therapy applications including the sustained correction of hAAT deficiency,” added Wilson.
The long life of the white blood cells sustaining the therapy was a major surprise of the study.
Emphysema or COPD is a progressive lung disease that causes severe shortness of breath. There is treatment available, but no cure.
People born with Alpha-1 (a genetic mutation that causes a deficiency in alpha-1 antitrypsin in the blood) are predisposed to COPD as well as cirrhosis of the liver.
