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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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Detalles Bibliográficos
Autores principales: Sutter, Marco A., Cremona, Tiziana P., Nita, Izabela, Cavarra, Eleonora, Lungarella, Giuseppe, Lewis, Eli C., Schittny, Johannes C., Geiser, Thomas, Gazdhar, Amiq
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7559762/
https://www.ncbi.nlm.nih.gov/pubmed/32825773
http://dx.doi.org/10.3390/pharmaceutics12090793