In utero gene editing for monogenic lung disease

Monogenic lung diseases that are caused by mutations in surfactant genes of the pulmonary epithelium are marked by perinatal lethal respiratory failure or chronic diffuse parenchymal lung disease with few therapeutic options. Using a CRISPR fluorescent reporter system, we demonstrate that precisely...

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Bibliografiska uppgifter
I publikationen:Sci Transl Med
Huvudupphovsmän: Alapati, Deepthi, Zacharias, William J., Hartman, Heather A., Rossidis, Avery C., Stratigis, John D., Ahn, Nicholas J, Coons, Barbara, Zhou, Su, Li, Hiaying, Singh, Kshitiz, Katzen, Jeremy, Tomer, Yaniv, Chadwick, Alexandra C., Musunuru, Kiran, Beers, Michael F., Morrisey, Edward E., Peranteau, William H.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2019
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC6822403/
https://ncbi.nlm.nih.gov/pubmed/30996081
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aav8375
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