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

Surfactant protein B (SP-B) deficiency is an autosomal recessive disorder that impairs surfactant homeostasis and manifests as lethal respiratory distress. A compelling argument exists for gene therapy to treat this disease, as de novo protein synthesis of SP-B in alveolar type 2 epithelial cells is...

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Pubblicato in:Nat Commun
Autori principali: Kang, Martin H., van Lieshout, Laura P., Xu, Liqun, Domm, Jakob M., Vadivel, Arul, Renesme, Laurent, Mühlfeld, Christian, Hurskainen, Maria, Mižíková, Ivana, Pei, Yanlong, van Vloten, Jacob P., Thomas, Sylvia P., Milazzo, Claudia, Cyr-Depauw, Chanèle, Whitsett, Jeffrey A., Nogee, Lawrence M., Wootton, Sarah K., Thébaud, Bernard
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2020
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7414154/
https://ncbi.nlm.nih.gov/pubmed/32764559
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17577-8
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