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Article

In utero gene editing for monogenic lung disease.

Science translational medicine - 17 Apr 2019

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

Abstract excerpt

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 timed in utero intra-amniotic delivery of CRISPR-Cas9 gene editing reagents during fetal development results in...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Pulmonary Surfactant-Associated Protein C

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Identifiers

PMID: 30996081
DOI: 10.1126/scitranslmed.aav8375
PMCID: PMC6822403

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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