A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency

A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency

Surfactant protein B (SP-B) deficiency is a genetic lung disease that results in lethal respiratory distress within months of birth. Here, the authors describe a gene therapy strategy using a rationally designed AAV6 capsid that restores surfactant homeostasis, prevents lung injury, and improves sur...

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Autores principales: Martin H. Kang, Laura P. van Lieshout, Liqun Xu, Jakob M. Domm, Arul Vadivel, Laurent Renesme, Christian Mühlfeld, Maria Hurskainen, Ivana Mižíková, Yanlong Pei, Jacob P. van Vloten, Sylvia P. Thomas, Claudia Milazzo, Chanèle Cyr-Depauw, Jeffrey A. Whitsett, Lawrence M. Nogee, Sarah K. Wootton, Bernard Thébaud
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
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Science
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Acceso en línea:https://doaj.org/article/6438f94f53b5451b8361dfa466593f2a
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https://doaj.org/article/6438f94f53b5451b8361dfa466593f2a

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