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

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

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...

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Bibliographic Details
Main Authors: Marco A. Sutter, Tiziana P. Cremona, Izabela Nita, Eleonora Cavarra, Giuseppe Lungarella, Eli C. Lewis, Johannes C. Schittny, Thomas Geiser, Amiq Gazdhar
Format: Article
Language:English
Published: MDPI AG 2020-08-01
Series:Pharmaceutics
Subjects:
alpha1 antitrypsin
nonviral gene delivery
electroporation
localized gene delivery
emphysema
liver gene transfer
Online Access:https://www.mdpi.com/1999-4923/12/9/793

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https://www.mdpi.com/1999-4923/12/9/793

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