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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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Detalhes bibliográficos
Publicado no:Pharmaceutics
Main Authors: Sutter, Marco A., Cremona, Tiziana P., Nita, Izabela, Cavarra, Eleonora, Lungarella, Giuseppe, Lewis, Eli C., Schittny, Johannes C., Geiser, Thomas, Gazdhar, Amiq
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2020
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7559762/
https://ncbi.nlm.nih.gov/pubmed/32825773
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/pharmaceutics12090793
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