Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

α1-antitrypsin deficiency is a rare genetic condition that can cause liver and/or lung disease. There is currently no cure for this disorder, although repeated infusions of plasma-purified protein may slow down emphysema progression. Gene therapy in which a single recombinant adeno-associated viral...

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Egile Nagusiak:	Marina Zieger, Florie Borel, Cynthia Greer, Gwladys Gernoux, Meghan Blackwood, Terence R. Flotte, Christian Mueller
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	Elsevier 2022-06-01
Saila:	Molecular Therapy: Methods & Clinical Development
Gaiak:	AAV COPD α1-antitrypsin deficiency genetic lung disease emphysema SERPINA1
Sarrera elektronikoa:	http://www.sciencedirect.com/science/article/pii/S232905012200050X
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