2020
DOI: 10.3390/pharmaceutics12090793
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In Vivo Electroporation-Mediated, Intrahepatic Alpha1 Antitrypsin Gene Transfer Reduces Pulmonary Emphysema in Pallid Mice

Abstract: 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 therapeutic levels of circulating AAT using nonviral in vivo electroporation-mediated gene transfer to the liver. Methods: In vivo intrahepatic electroporation-mediated human AAT gene transfer was performed in C57 Bl/6… Show more

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“… 21 Recently, liver electroporation has been used for the expression of α1-antitrypsin (AAT) in AAT-deficient mice, resulting in a reduction of pulmonary emphysema. 22 Transcutaneous ultrasound has been proven to be efficient for the delivery of genetic material to the liver of mice and pigs. 23 , 24 So far, the use of these approaches in liver-directed GT is still restricted to experimental animal studies.…”
Section: Non-viral Vectorsmentioning
confidence: 99%
“… 21 Recently, liver electroporation has been used for the expression of α1-antitrypsin (AAT) in AAT-deficient mice, resulting in a reduction of pulmonary emphysema. 22 Transcutaneous ultrasound has been proven to be efficient for the delivery of genetic material to the liver of mice and pigs. 23 , 24 So far, the use of these approaches in liver-directed GT is still restricted to experimental animal studies.…”
Section: Non-viral Vectorsmentioning
confidence: 99%