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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Nature Portfolio
2020
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oai:doaj.org-article:6438f94f53b5451b8361dfa466593f2a2021-12-02T17:06:35ZA lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency10.1038/s41467-020-17577-82041-1723https://doaj.org/article/6438f94f53b5451b8361dfa466593f2a2020-08-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-17577-8https://doaj.org/toc/2041-1723Surfactant 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 survival in a mouse model of SP-B deficiency.Martin H. KangLaura P. van LieshoutLiqun XuJakob M. DommArul VadivelLaurent RenesmeChristian MühlfeldMaria HurskainenIvana MižíkováYanlong PeiJacob P. van VlotenSylvia P. ThomasClaudia MilazzoChanèle Cyr-DepauwJeffrey A. WhitsettLawrence M. NogeeSarah K. WoottonBernard ThébaudNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-20 (2020) |
| institution |
DOAJ |
| collection |
DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q 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 A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| description |
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 survival in a mouse model of SP-B deficiency. |
| format |
article |
| author |
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 |
| author_facet |
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 |
| author_sort |
Martin H. Kang |
| title |
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| title_short |
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| title_full |
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| title_fullStr |
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| title_full_unstemmed |
A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency |
| title_sort |
lung tropic aav vector improves survival in a mouse model of surfactant b deficiency |
| publisher |
Nature Portfolio |
| publishDate |
2020 |
| url |
https://doaj.org/article/6438f94f53b5451b8361dfa466593f2a |
| work_keys_str_mv |
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