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

α(1)-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 vira...

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Detalles Bibliográficos
Autores principales: Zieger, Marina, Borel, Florie, Greer, Cynthia, Gernoux, Gwladys, Blackwood, Meghan, Flotte, Terence R., Mueller, Christian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9097330/
https://www.ncbi.nlm.nih.gov/pubmed/35592360
http://dx.doi.org/10.1016/j.omtm.2022.04.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9097330/
https://www.ncbi.nlm.nih.gov/pubmed/35592360
http://dx.doi.org/10.1016/j.omtm.2022.04.003

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