Targeted Gene Therapy Could Potentially Treat Emphysema

After lung-targeted gene therapy, patients produced normal alpha-1-antityrpsin.

A novel targeted gene therapy could potentially improve treatment for lung diseases, such as emphysema.

Alpha-1-antityrpsin deficiency (AATD), a common genetic cause of emphysema, is caused by the inheritance of a mutated AAT gene. Currently, the treatment of patients with AAT includes intravenous infusions of non-mutated AAT proteins, according to the study published by Molecular Therapy -- Methods & Clinical Development.

Patients with AATD have particularly low levels of AAT in their lungs.

In the study, researchers were able to deliver a copy of normal AAT to the lungs. They discovered that this normal gene existed in lung cells for at least 1 year.

Researchers also found that multiple types of lung cells and liver cells are affected by the treatment, and normal AAT was produced. The amount of normal AAT created was able to decrease the severity of the lung disease, according to the study.

"These results support direct transgene delivery to the lung as a potential alternative approach to achieve the goal of developing a gene therapy for AATD," concluded corresponding author Andrew Wilson, MD.