In Vivo Electroporation-Mediated, Intrahepatic Alpha1 Antitrypsin Gene Transfer Reduces Pulmonary Emphysema in Pallid Mice

• Main Authors: Marco A. Sutter (Author), Tiziana P. Cremona (Author), Izabela Nita (Author), Eleonora Cavarra (Author), Giuseppe Lungarella (Author), Eli C. Lewis (Author), Johannes C. Schittny (Author), Thomas Geiser (Author), Amiq Gazdhar (Author)
• Format: Book
• Published: MDPI AG, 2020-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Rationale: Mutation in the alpha1 antitrypsin (AAT) gene leads to low circulating levels of AAT, which is associated with several disease processes including pulmonary emphysema. The standard of care relies on substitution with plasma-purified AAT. We studied a novel approach to obtain sustained the...