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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Bibliographic Details
Published in:Sci Transl Med
Main Authors: 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.
Format: Artigo
Language:Inglês
Published: 2019
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Article
Online Access: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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